https://www.trialfriend.com/blog Rare disease news for patients: FDA decisions, clinical trial results, gene therapy access and drug pipelines, sourced from the FDA and ClinicalTrials.gov. en-us Tue, 29 Sep 2026 12:00:00 GMT https://www.trialfriend.com/og-image.png https://www.trialfriend.com/blog https://www.trialfriend.com/blog/atacicept-vs-povetacicept-iga-nephropathy https://www.trialfriend.com/blog/atacicept-vs-povetacicept-iga-nephropathy Jason Hunter Tue, 29 Sep 2026 12:00:00 GMT Atacicept and povetacicept block the same 2 immune signals in IgA nephropathy. Atacicept is approved as Trutakna and now has 2-year kidney data. Povetacicept faces an FDA decision on November 30th and would be injected 13 times a year instead of 52. A side-by-side table, the trial results, what an independent review found, what patients told the FDA, and 7 real-world situations. Guide IgA Nephropathy Treatment Comparison Clinical Trials A man in a grey T-shirt holds an injection pen near his stomach, about to give himself a dose at home. https://www.trialfriend.com/blog/rare-disease-clinical-trials-medication-exclusions-2026 https://www.trialfriend.com/blog/rare-disease-clinical-trials-medication-exclusions-2026 Jason Hunter Mon, 28 Sep 2026 12:00:00 GMT We ran every open treatment trial for 37 rare diseases through Trial Friend's medication checker. Of 1,853 trials, 617 name a medication that disease's patients commonly take in their exclusion rules, and nearly 1 in 4 of the time windows they set reaches back about 6 months or longer. Data Clinical Trials Eligibility Medications A hand takes a pill from a weekly pill organizer on a dark table, next to a blister pack. https://www.trialfriend.com/blog/alpha-1-antitrypsin-deficiency-treatments-compared https://www.trialfriend.com/blog/alpha-1-antitrypsin-deficiency-treatments-compared Jason Hunter Sun, 27 Sep 2026 12:00:00 GMT Every FDA-approved alpha-1 antitrypsin deficiency treatment is the same idea: a weekly IV infusion of AAT protein from donated plasma. Here is how Prolastin-C, Aralast NP, Zemaira and Glassia compare, what the largest placebo-controlled trial actually showed, why none of them treats the liver, and where the pipeline stands, from a monthly lab-made AAT to a one-time base edit that lifted AAT above the historical 11 micromolar target. Guide Alpha-1 Antitrypsin Deficiency Treatment Comparison Gene Editing Clinical Trials A young man in a grey hoodie stretches his arms on a sunlit forest path before a run. https://www.trialfriend.com/blog/insurance-denied-rare-disease-drug-how-to-appeal https://www.trialfriend.com/blog/insurance-denied-rare-disease-drug-how-to-appeal Jason Hunter Sat, 26 Sep 2026 12:00:00 GMT 2026-09-27 Insurers denied 1 in 5 in-network claims in 2023 and fewer than 1% were appealed, yet insurers reversed themselves on 44% of the appeals they did get. This guide walks through every deadline for employer plans, marketplace plans, Medicare and Medicaid, the 6 denial scenarios rare disease patients hit most, what to put in the appeal letter, and who will do it with you for free. Guide Insurance Patient Assistance Medicare Prior Authorization A man in a blue shirt reads a letter at his kitchen table, mouth open in surprise, next to a blue mug and a plate of fruit. https://www.trialfriend.com/blog/attr-amyloidosis-treatments-compared https://www.trialfriend.com/blog/attr-amyloidosis-treatments-compared Jason Hunter Fri, 25 Sep 2026 12:00:00 GMT 2026-09-29 Transthyretin is a protein the liver makes, and in ATTR amyloidosis it misfolds and piles up in the heart and nerves. The FDA has approved 6 drugs for it since 2018, and 5 are still sold. Stabilizers (2 of them) hold the TTR protein together; silencers (the other 3) stop the liver from making it. Here is what each one showed in its own trial, what a year of treatment actually looks like (1,460 tablets versus 4 injections), why one was discontinued, and what the trial that failed in July 2026 taught doctors about combining them. Guide ATTR Amyloidosis Cardiology Treatment Comparison FDA A doctor in a white coat talks with an older man with grey hair as they sit together on a couch. https://www.trialfriend.com/blog/medicare-open-enrollment-2027-rare-disease-drugs https://www.trialfriend.com/blog/medicare-open-enrollment-2027-rare-disease-drugs Jason Hunter Fri, 25 Sep 2026 12:00:00 GMT Medicare open enrollment runs October 15th to December 7th, 2026. For anyone on a rare disease drug, the 2027 Part D cap rises to $2,400, copay cards still don't apply, and infused drugs follow different rules. What to check before you switch or stay. Guide Medicare Insurance Patient Assistance Cost Three older adults at a table, one pointing at a form while another fills it in. https://www.trialfriend.com/blog/ino-3107-rrp-fda-decision-october-2026 https://www.trialfriend.com/blog/ino-3107-rrp-fda-decision-october-2026 Jason Hunter Thu, 24 Sep 2026 12:00:00 GMT For decades, the only answer to recurrent respiratory papillomatosis was another surgery. The FDA approved the first drug in August 2025, and on October 30th it is due to decide on a second, INOVIO's INO-3107. What the trials showed, the accelerated approval question hanging over the review, and the 8 RRP studies open now. News Recurrent Respiratory Papillomatosis FDA INO-3107 A doctor presses a stethoscope to a patient's chest to listen to their breathing. https://www.trialfriend.com/blog/acromegaly-clinical-trials-2026-open-studies https://www.trialfriend.com/blog/acromegaly-clinical-trials-2026-open-studies Jason Hunter Thu, 24 Sep 2026 12:00:00 GMT As of September 23rd, 17 acromegaly studies are recruiting or about to open worldwide, and only 3 have US sites that enroll patients. Here is what each one tests, where the sites are, how well today's treatments worked in their own trials, and what a skeleton from 1783 taught doctors about who should get a genetic test. Guide Acromegaly Clinical Trials FDA Two doctors hold up a sheet of brain MRI scans against a bright window to review them. https://www.trialfriend.com/blog/lirafugratinib-fgfr2-cholangiocarcinoma-fda-decision https://www.trialfriend.com/blog/lirafugratinib-fgfr2-cholangiocarcinoma-fda-decision Jason Hunter Wed, 23 Sep 2026 12:00:00 GMT 2026-09-29 The FDA approved lirafugratinib as Lyrfigtu on September 23rd, 2 days early, for previously treated cholangiocarcinoma with an FGFR2 fusion. It is the third FGFR drug available for this cancer and the first built to block FGFR2 alone. What the 116-patient ReFocus cohort showed, what the label warns about, how it compares with the drugs before it, and what new research says about using it in sequence. News Cholangiocarcinoma FDA Targeted Therapy Lyrfigtu An open palm holding 3 different capsules against a dark background. https://www.trialfriend.com/blog/camzyos-adolescent-hcm-fda-decision-september-30-2026 https://www.trialfriend.com/blog/camzyos-adolescent-hcm-fda-decision-september-30-2026 Jason Hunter Tue, 22 Sep 2026 12:00:00 GMT The FDA is due to decide by September 30, 2026 whether Camzyos (mavacamten) can be prescribed to adolescents aged 12 to 17 with symptomatic obstructive hypertrophic cardiomyopathy. It would be the first drug approved for HCM in anyone under 18. What the 44-patient SCOUT-HCM trial showed, what the boxed warning means for a teenager, and what changes for families if the answer is yes. News Hypertrophic Cardiomyopathy FDA Overhead view of a group of runners on a red track, with one runner falling behind the pack. https://www.trialfriend.com/blog/aqneursa-ataxia-telangiectasia-first-fda-approved-treatment https://www.trialfriend.com/blog/aqneursa-ataxia-telangiectasia-first-fda-approved-treatment Jason Hunter Mon, 21 Sep 2026 12:00:00 GMT 2026-09-26 On September 18th the FDA approved Aqneursa (levacetylleucine) for the ataxia of ataxia-telangiectasia, the first drug approved for A-T anywhere in the world. It comes less than 8 months after the largest A-T trial ever run failed. What the 73-patient study showed, what the drug does and does not treat, and why families can get it this week rather than next year. News Ataxia-Telangiectasia FDA A toddler walking across grass while holding a parent's hand on each side. https://www.trialfriend.com/blog/emcitate-tiratricol-mct8-deficiency-fda-decision https://www.trialfriend.com/blog/emcitate-tiratricol-mct8-deficiency-fda-decision Jason Hunter Fri, 18 Sep 2026 12:00:00 GMT 2026-09-28 The FDA approved Emcitate (tiratricol) on September 28, 2026, the first treatment for MCT8 deficiency, a disease where thyroid hormone floods the body and never reaches the brain. What the US label says, what 3 trials found, what the drug does and does not fix, and how US families can get it. News MCT8 Deficiency FDA A newborn lying on his stomach and lifting his head to look to the side. https://www.trialfriend.com/blog/isembyld-apitegromab-sma-approval-muscle-targeted https://www.trialfriend.com/blog/isembyld-apitegromab-sma-approval-muscle-targeted Jason Hunter Thu, 17 Sep 2026 12:00:00 GMT 2026-09-29 The FDA approved Isembyld on September 11th, 19 days ahead of its deadline. It is the first SMA drug that treats muscle rather than motor neurons, and the label requires you to already be on Spinraza or Evrysdi. Here is what it does, who qualifies, and the fracture risk nobody is leading with. News Spinal Muscular Atrophy Treatment Access A young patient steadying herself on parallel bars while a physical therapist records her motor function assessment on a clipboard. https://www.trialfriend.com/blog/ux111-sanfilippo-gene-therapy-fda-decision https://www.trialfriend.com/blog/ux111-sanfilippo-gene-therapy-fda-decision Jason Hunter Thu, 17 Sep 2026 12:00:00 GMT 2026-09-25 The FDA approved UX111 on September 17th, 2 days early, under the brand name Fayuvi, as the first treatment in the history of Sanfilippo syndrome. It was a full approval on a cognitive endpoint, not the biomarker-based one everyone expected. Here is who the label covers, how old the trial children were, and the 5 warnings on it. News Gene Therapy Sanfilippo Syndrome MPS III FDA UX111 Fayuvi A parent's hands gently cradling a young child's hand, reflecting the families awaiting the FDA's decision on the first Sanfilippo syndrome treatment. https://www.trialfriend.com/blog/iga-nephropathy-approved-drugs-what-labels-promise https://www.trialfriend.com/blog/iga-nephropathy-approved-drugs-what-labels-promise Jason Hunter Wed, 16 Sep 2026 12:00:00 GMT 2026-09-25 IgA nephropathy went from zero approved drugs to 6 in 5 years. 3 have proven they slow kidney function decline. The other 3 are approved on a urine protein number and still owe that proof. Here is the difference and why it matters to you. Guide IgA Nephropathy Treatment Access A gloved hand lifting a labeled specimen tube from a rack in a clinical laboratory. https://www.trialfriend.com/blog/alpha-gal-syndrome-blood-transfusion-risk-type-o https://www.trialfriend.com/blog/alpha-gal-syndrome-blood-transfusion-risk-type-o Jason Hunter Wed, 16 Sep 2026 12:00:00 GMT New national blood bank guidance describes transfusion-related alpha-gal syndrome, where people sensitized by tick bites react to platelets and plasma from type B donors. 9 published cases, all in type O patients, 1 fatal. Here is what to say before surgery. News Alpha-Gal Syndrome Medication Safety A bag of donated blood hanging on a line above a drip chamber during a transfusion. https://www.trialfriend.com/blog/palsonify-acromegaly-pill-one-year-switching https://www.trialfriend.com/blog/palsonify-acromegaly-pill-one-year-switching Jason Hunter Tue, 15 Sep 2026 12:00:00 GMT Palsonify turns 1 on September 25th. A year of real-world data shows what trading monthly injections for a daily tablet actually involves, from the empty stomach rule to the $290,000 question insurers are mostly saying yes to. News Acromegaly Treatment Access An open hand holding pills next to a weekly pill organizer and a glass of water on a light blue surface. https://www.trialfriend.com/blog/free-alpha-gal-medication-checker-launch https://www.trialfriend.com/blog/free-alpha-gal-medication-checker-launch Jason Hunter Tue, 15 Sep 2026 12:00:00 GMT 2026-09-28 Type any drug name and see which manufacturers' versions contain gelatin, heparin, or other mammalian-derived ingredients, screened directly from FDA labels. Free, no login, and honest about what it cannot tell you. News Alpha-Gal Syndrome Tools Medication Safety Green and white capsules sealed in clear blister packs on a teal background. https://www.trialfriend.com/blog/fda-approval-to-first-dose-rare-disease-timeline https://www.trialfriend.com/blog/fda-approval-to-first-dose-rare-disease-timeline Jason Hunter Mon, 14 Sep 2026 12:00:00 GMT 2026-09-17 Approval day feels like the finish line. It is the starting gun for a second race through specialty pharmacies, insurance reviews, and copay programs. Real timelines from 5 recent rare disease launches, and the moves that shorten the wait. Guide FDA Insurance Drug Access Calendar grid with weeks of days filled in teal, counting toward one day marked in amber. https://www.trialfriend.com/blog/when-a-clinical-trial-fails-angelman-dm1 https://www.trialfriend.com/blog/when-a-clinical-trial-fails-angelman-dm1 Jason Hunter Mon, 14 Sep 2026 12:00:00 GMT In the space of 6 days, Phase 3 trials failed in Angelman syndrome and myotonic dystrophy type 1, two diseases with no approved treatment. The coverage has been about stock prices. This is about the families who enrolled, what happens to them now, and what is honestly left to hope for. News Angelman Syndrome Myotonic Dystrophy Clinical Trials Two people holding hands in comfort, one resting a hand on the other's. https://www.trialfriend.com/blog/rare-disease-trial-travel-burden-2026 https://www.trialfriend.com/blog/rare-disease-trial-travel-burden-2026 Jason Hunter Sat, 12 Sep 2026 12:00:00 GMT We mapped all 20,732 US study sites behind the 6,372 recruiting trials across the 195 rare diseases Trial Friend tracks. More than half of the trials recruiting on US soil do it at a single site, 56% of recruiting trials worldwide have no US site at all, and for a patient in Wyoming the numbers get much worse. Data Clinical Trials Travel Access Empty two-lane desert highway running straight toward distant mountains. https://www.trialfriend.com/blog/fda-clinical-hold-what-it-means-for-patients https://www.trialfriend.com/blog/fda-clinical-hold-what-it-means-for-patients Jason Hunter Sat, 12 Sep 2026 12:00:00 GMT On September 4th the FDA paused new enrollment in Biohaven's opakalim epilepsy trials over a safety signal from rodent lab studies. Headlines said "hold." Patients heard "over." Here is what a clinical hold actually is, how often holds lift, what happened in famous cases, and the questions to ask if it happens to your trial. Guide FDA Clinical Trials Safety FDA headquarters sign in front of the agency's White Oak campus in Maryland. https://www.trialfriend.com/blog/zilurgisertib-fop-fda-decision-september-2026 https://www.trialfriend.com/blog/zilurgisertib-fop-fda-decision-september-2026 Jason Hunter Fri, 11 Sep 2026 12:00:00 GMT 2026-09-26 FOP went centuries without a single approved treatment. Now the ultra-rare bone disease could see its third in barely 3 years when the FDA rules on zilurgisertib, a once-daily pill from Mirum and Incyte, on September 26th. News FOP FDA Zilurgisertib X-ray of a human torso and shoulders, the kind of imaging used to track new bone growth in FOP. https://www.trialfriend.com/blog/rare-disease-clinical-trials-2026-census https://www.trialfriend.com/blog/rare-disease-clinical-trials-2026-census Jason Hunter Wed, 09 Sep 2026 12:00:00 GMT 2026-09-28 We counted every active clinical trial across the 195 rare diseases Trial Friend tracks: 9,501 studies, 5,547 recruiting, 6 diseases with nothing at all. Add 7 approvals already this year and 21 FDA decisions still to come, and 2026 is shaping up as a landmark year with an uncomfortable asterisk. Data Clinical Trials FDA Research Fluorescence microscope view of cells glowing purple, blue, and green against a black background. https://www.trialfriend.com/blog/september-2026-rare-disease-fda-decisions https://www.trialfriend.com/blog/september-2026-rare-disease-fda-decisions Jason Hunter Tue, 08 Sep 2026 12:00:00 GMT 2026-09-28 Between September 19th and September 30th, the FDA is due to rule on 8 rare disease therapies. Sanfilippo syndrome, Alexander disease, ataxia-telangiectasia, and MCT8 deficiency have never had an approved treatment. That could change within 3 weeks. News FDA Sanfilippo Syndrome Alexander Disease SMA FOP A hand holds an hourglass with sand running through it, marking the final weeks before 8 rare disease FDA decisions in September 2026. https://www.trialfriend.com/blog/kv7-epilepsy-race-opakalim-azetukalner https://www.trialfriend.com/blog/kv7-epilepsy-race-opakalim-azetukalner Jason Hunter Wed, 26 Aug 2026 12:00:00 GMT 2026-09-23 Nine years after ezogabine left the market under a cloud of blue-gray skin and retinal warnings, two companies are one readout away from bringing its mechanism back. Xenon's azetukalner posted the strongest Phase 3 focal seizure data in years. On August 26, the maker of Xcopri paid $350 million upfront for Biohaven's opakalim without waiting to see the data. News Epilepsy Focal Epilepsy Clinical Trials Chemical structure of ezogabine, the Kv7-opening seizure drug withdrawn from the market in 2017. https://www.trialfriend.com/blog/marrvel-mcp-ai-rare-disease-variant-interpretation https://www.trialfriend.com/blog/marrvel-mcp-ai-rare-disease-variant-interpretation Jason Hunter Mon, 25 May 2026 12:00:00 GMT A team at Baylor College of Medicine and Texas Children's Hospital just published a tool that lets a small, locally-runnable AI model interpret genetic variants for Mendelian rare diseases at 94% accuracy, up from 41% without it. The tool is open source, publicly hosted, and built on the Model Context Protocol that Anthropic released in 2024. For patients sitting on a variant of uncertain significance, this is the most important rare disease AI story of the year. News Rare Disease Genetic Testing AI Diagnosis Stylized DNA helix glowing across a dark background, representing AI-assisted rare disease variant interpretation. https://www.trialfriend.com/blog/rare-disease-incidental-findings-diagnosed-by-accident https://www.trialfriend.com/blog/rare-disease-incidental-findings-diagnosed-by-accident Jason Hunter Wed, 20 May 2026 12:00:00 GMT 2026-09-29 Some of the most consequential rare disease diagnoses started with a routine carpal tunnel surgery, an unrelated CT scan, a yearly eye exam, or a young person's first stroke. This is a clinically documented guide to the incidental findings that have surfaced ATTR amyloidosis, Wilson disease, pheochromocytoma, Fabry disease, hereditary hemorrhagic telangiectasia, and other rare conditions years before symptoms made them obvious. Guide Rare Disease Diagnosis Incidental Findings Patient Stories A close-up photograph of a medical imaging scan or specialist examination, representing the routine clinical encounters where rare diseases are often unexpectedly discovered before symptoms make them clinically obvious https://www.trialfriend.com/blog/lone-star-tick-diseases-alpha-gal-syndrome-heartland-virus https://www.trialfriend.com/blog/lone-star-tick-diseases-alpha-gal-syndrome-heartland-virus Jason Hunter Tue, 12 May 2026 12:00:00 GMT The lone star tick is the small white-dotted tick driving the surge in Alpha-Gal Syndrome cases across the United States. Range is expanding north and west, and the same tick carries ehrlichiosis, Heartland virus, Bourbon virus, STARI, and tularemia. This is the comprehensive guide to identifying lone star ticks, the diseases they cause, what to do after a bite, and how to prevent the next one. Guide Alpha-Gal Syndrome Tick-Borne Disease Lone Star Tick Prevention A high-resolution photograph of an adult female lone star tick (Amblyomma americanum) resting on a green leaf, with the distinctive white dot clearly visible in the center of her dark reddish-brown back. This is the tick species responsible for Alpha-Gal Syndrome and several other rare tick-borne diseases in the United States. Image credit: Jim Gathany, Centers for Disease Control and Prevention Public Health Image Library, public domain. https://www.trialfriend.com/blog/rystiggo-myasthenia-gravis-fcrn-inhibitor-mycaring-trial https://www.trialfriend.com/blog/rystiggo-myasthenia-gravis-fcrn-inhibitor-mycaring-trial Jason Hunter Sat, 09 May 2026 12:00:00 GMT 2026-09-25 Rystiggo (rozanolixizumab-noli) is an FcRn inhibitor approved for adults with anti-AChR or anti-MuSK antibody-positive generalized myasthenia gravis. This guide covers how it works, the MycarinG Phase 3 trial that got it approved, the side effect profile from the actual data, how it compares to Vyvgart and Imaavy, the pipeline drugs coming behind it, and how patients access it. Guide Myasthenia Gravis FcRn Inhibitor Rare Autoimmune Rystiggo A close-up photograph of a person's face showing asymmetric ptosis, with the right eyelid drooping noticeably while the left eye is fully open, illustrating the classic ocular sign of generalized myasthenia gravis that Rystiggo (rozanolixizumab) is approved to treat in anti-AChR or anti-MuSK antibody-positive adults https://www.trialfriend.com/blog/alpha-gal-syndrome-medications-vaccines-medical-products-to-avoid https://www.trialfriend.com/blog/alpha-gal-syndrome-medications-vaccines-medical-products-to-avoid Jason Hunter Fri, 08 May 2026 12:00:00 GMT 2026-09-28 Alpha-Gal Syndrome (AGS) is more than a red meat allergy. Heparin, certain monoclonal antibodies, gelatin in vaccines and capsules, surgical glues, and many over-the-counter medications can contain mammalian-derived alpha-gal and trigger a reaction. This is the comprehensive list of hidden alpha-gal exposures in healthcare, with the products to ask about, the safer alternatives, and what to tell every doctor before any procedure. Guide Alpha-Gal Syndrome Tick-Borne Disease Medication Safety Allergy A vial of Adrenalin (epinephrine injection, USP, 1 mg/ml) and a syringe resting on a printed page with bold text reading Alpha-gal allergy, also referenced as Mammalian Meat Allergy and galactose-alpha, illustrating the emergency-preparedness materials Alpha-Gal Syndrome patients carry to manage potential anaphylactic reactions to hidden mammalian-derived medications and products https://www.trialfriend.com/blog/casgevy-vs-lyfgenia-sickle-cell-gene-therapy-comparison https://www.trialfriend.com/blog/casgevy-vs-lyfgenia-sickle-cell-gene-therapy-comparison Jason Hunter Thu, 07 May 2026 12:00:00 GMT 2026-09-29 Casgevy and Lyfgenia are the two FDA-approved gene therapies for sickle cell disease, both approved on the same day in December 2023. They use different biology, have different safety profiles (Lyfgenia carries a boxed warning), and cost different amounts. Here's how they compare and how to think about the decision with your hematologist. Guide Sickle Cell Disease Gene Therapy Casgevy Lyfgenia A 3D illustration of red blood cells inside a blood vessel, showing normal disc-shaped cells alongside elongated, crescent-shaped sickled cells clumping together, illustrating the underlying cellular biology that Casgevy and Lyfgenia gene therapies target https://www.trialfriend.com/blog/school-accommodations-rare-disease-parent-guide https://www.trialfriend.com/blog/school-accommodations-rare-disease-parent-guide Jason Hunter Wed, 06 May 2026 12:00:00 GMT A practical guide for U.S. parents of children with rare diseases. Covers the differences between 504 plans, IEPs, and Individual Health Care Plans, the accommodations that actually get approved for fatigue, infection risk, mobility, cognitive load, and medical care during the day, the parent mistakes that cost families months, and the college transition where the rules quietly change. Guide Pediatric Care School Accommodations 504 Plan IEP A black spiral notebook with the words Individualized Education Program in white lettering, surrounded by brightly colored wooden block pieces and a yellow alarm clock on a coral background, illustrating the planning, advocacy, and timing involved in setting up an IEP or 504 plan https://www.trialfriend.com/blog/stacked-diagnoses-eds-mcas-pots-mthfr-tick-borne https://www.trialfriend.com/blog/stacked-diagnoses-eds-mcas-pots-mthfr-tick-borne Jason Hunter Sat, 02 May 2026 12:00:00 GMT Some rare disease patients walk into a specialist's office for one diagnosis and walk out, eventually, with 5. The hEDS / MCAS / POTS triad now has formal academic recognition. Layered with tick-borne coinfections and an MTHFR variant, it becomes one of the most complex and most missed presentations in medicine. What's solidly evidenced, what's still debated, and what patients can actually do. Guide Ehlers-Danlos Syndrome MCAS POTS Tick-Borne Disease MTHFR Layered passage of colorful vertical slats forming a tunnel of overlapping hues, representing how multiple rare conditions stack and interconnect into a single complex clinical picture https://www.trialfriend.com/blog/heparin-induced-thrombocytopenia-cad-1005-phase-3-2026 https://www.trialfriend.com/blog/heparin-induced-thrombocytopenia-cad-1005-phase-3-2026 Jason Hunter Thu, 30 Apr 2026 12:00:00 GMT On April 30, 2026, Cadrenal Therapeutics announced an FDA-cleared path to a pivotal Phase 3 trial for CAD-1005 in heparin-induced thrombocytopenia. If approved, it would be the first new drug specifically developed for HIT since argatroban in 2000. HIT kills more than 1 in 5 patients in some studies. Here's why a new option matters and what's coming next. News Heparin-Induced Thrombocytopenia Clinical Trials Anticoagulation Rare Disease Empty hospital procedure room with surgical light, IV pole, and adjustable bed, representing the inpatient setting where heparin-induced thrombocytopenia develops and is treated https://www.trialfriend.com/blog/lonvo-z-hae-haelo-gene-editing-results https://www.trialfriend.com/blog/lonvo-z-hae-haelo-gene-editing-results Jason Hunter Thu, 30 Apr 2026 12:00:00 GMT 2026-09-29 On April 27, 2026, Intellia Therapeutics reported positive Phase 3 results for lonvo-z in hereditary angioedema. A single infusion cut HAE attacks by 87%, with 62% of treated patients going six months attack-free and therapy-free. It's the first Phase 3 win for any in vivo CRISPR therapy. For HAE patients living with chronic injections and unpredictable attacks, the implications are real. News Gene Therapy Hereditary Angioedema CRISPR Clinical Trials Solitary figure on a rocky outcrop overlooking layered mountain ridges in golden morning light, representing the first regulatory crossing for in vivo gene editing https://www.trialfriend.com/blog/diagnostic-odyssey-whole-genome-sequencing-rare-disease https://www.trialfriend.com/blog/diagnostic-odyssey-whole-genome-sequencing-rare-disease Jason Hunter Wed, 29 Apr 2026 12:00:00 GMT A rare disease patient in the US waits 6 years on average for a diagnosis, accumulates 17 medical encounters, and gets 2 to 3 wrong answers along the way. A test that finds the genetic cause in 5 days now costs $100 to run. Most patients still don't get it. Here's what's blocking the path and how to push through. Guide Diagnostic Odyssey Whole Genome Sequencing Rare Disease Insurance Aerial drone view of a long winding road cutting through a dense pine forest, representing the years-long search for a rare disease diagnosis https://www.trialfriend.com/blog/fda-plausible-mechanism-framework-rare-disease-gene-therapy https://www.trialfriend.com/blog/fda-plausible-mechanism-framework-rare-disease-gene-therapy Jason Hunter Fri, 24 Apr 2026 12:00:00 GMT A new FDA framework lets gene therapies and antisense treatments reach patients with ultra-rare diseases without massive clinical trials. 30 million Americans with rare diseases could eventually benefit. We break down what changed, who it helps first, and what the risks are. News Gene Therapy FDA CRISPR Rare Disease Policy DNA double helix representing gene editing therapies for rare diseases under the FDA plausible mechanism framework https://www.trialfriend.com/blog/rare-disease-emergency-room-what-er-doctors-dont-know https://www.trialfriend.com/blog/rare-disease-emergency-room-what-er-doctors-dont-know Jason Hunter Tue, 21 Apr 2026 12:00:00 GMT Emergency departments misdiagnose 1 in 18 patients. For rare disease patients, the odds are worse. Five conditions where the wrong ER response can turn a treatable crisis into a fatal one. Guide Rare Disease Emergency Medicine Patient Safety Sickle Cell Disease Hospital ward representing emergency room challenges for rare disease patients like sickle cell and Addison's disease https://www.trialfriend.com/blog/tick-borne-rare-diseases-lyme-coinfections https://www.trialfriend.com/blog/tick-borne-rare-diseases-lyme-coinfections Jason Hunter Sun, 19 Apr 2026 12:00:00 GMT The blacklegged tick carries at least 7 human pathogens. Three of them, babesiosis, bartonellosis, and anaplasmosis, are classified as rare diseases. Most doctors don't test for them. Here's what the science actually says. Guide Tick-Borne Disease Rare Disease Lyme Disease Babesiosis Bartonella Anaplasmosis Detailed illustration of a blacklegged tick, the primary vector for Lyme disease, babesiosis, and anaplasmosis https://www.trialfriend.com/blog/how-to-read-clinicaltrials-gov-listing https://www.trialfriend.com/blog/how-to-read-clinicaltrials-gov-listing Jason Hunter Fri, 17 Apr 2026 12:00:00 GMT ClinicalTrials.gov listings are written for regulators, not patients. We walk through a real Duchenne muscular dystrophy gene therapy trial, section by section, and translate every field into plain language. Guide Clinical Trials ClinicalTrials.gov Duchenne Muscular Dystrophy Cipher wheel representing how to decode and read a ClinicalTrials.gov listing for rare disease clinical trials https://www.trialfriend.com/blog/clinical-trial-participation-series https://www.trialfriend.com/blog/clinical-trial-participation-series Jason Hunter Thu, 16 Apr 2026 12:00:00 GMT A 4-part series covering everything patients and caregivers need to consider before, during, and after expressing interest in a clinical trial. From evaluating whether a trial fits your life, to talking with your doctor, to the questions that actually matter. Guide Clinical Trials Patient Resources Series Scientist using forceps in a clinical research laboratory setting https://www.trialfriend.com/blog/why-trial-friend-doesnt-recruit-patients https://www.trialfriend.com/blog/why-trial-friend-doesnt-recruit-patients Jason Hunter Wed, 15 Apr 2026 12:00:00 GMT Most clinical trial discovery tools exist to feed sponsor recruitment pipelines. That shapes what patients see, what gets ranked first, and where their data ends up. Here is why Trial Friend was built differently, and what 'patient-first' actually means once you get past the marketing copy. Perspective Patient Advocacy Clinical Trial Search Data Privacy Animated Trial Friend wordmark, morphing from Clinical Trial Finder https://www.trialfriend.com/blog/filspari-fsgs-fda-approval-april-2026 https://www.trialfriend.com/blog/filspari-fsgs-fda-approval-april-2026 Jason Hunter Tue, 14 Apr 2026 12:00:00 GMT 2026-09-25 On April 13, 2026, the FDA approved Filspari (sparsentan) for Focal Segmental Glomerulosclerosis, ending a 70-year wait for a disease-specific therapy. The story of how Travere Therapeutics got here, what the drug actually does, and how long patients will really wait to start treatment. News FSGS Kidney Disease FDA Approval Rare Disease Travere Therapeutics Filspari sparsentan FDA approval for FSGS focal segmental glomerulosclerosis rare kidney disease https://www.trialfriend.com/blog/rare-disease-trial-enrollment-slowdown-paradox https://www.trialfriend.com/blog/rare-disease-trial-enrollment-slowdown-paradox Jason Hunter Mon, 13 Apr 2026 12:00:00 GMT 2026-09-25 For decades, patients with rare diseases joined clinical trials because trials were the only option. Approved drugs have changed that math. The enrollment slowdown that has followed is reshaping how and where future therapies get developed. News Clinical Trials Rare Disease Drug Development Patient Decision Empty clinic chairs representing the rare disease clinical trial enrollment slowdown despite record FDA orphan drug approvals https://www.trialfriend.com/blog/how-to-appeal-insurance-denial-rare-disease-medication https://www.trialfriend.com/blog/how-to-appeal-insurance-denial-rare-disease-medication Jason Hunter Sat, 11 Apr 2026 12:00:00 GMT 2026-09-25 When a rare disease drug gets denied by insurance, most patients give up. The data says they shouldn't. A practical guide to prior authorizations, appeals, external reviews, and the people who can help. Guide Insurance Rare Disease Patient Advocacy Prior Authorization Person in a suit holding a torn piece of paper that reads Are You Covered, representing insurance coverage questions for rare disease patients https://www.trialfriend.com/blog/priority-review-vouchers-rare-disease-what-parents-should-know https://www.trialfriend.com/blog/priority-review-vouchers-rare-disease-what-parents-should-know Jason Hunter Thu, 09 Apr 2026 12:00:00 GMT Most parents of kids with rare diseases have never heard of priority review vouchers. This obscure FDA program is one of the biggest reasons any company bothered developing a treatment for their child's condition in the first place. Guide Rare Disease FDA Drug Development Illustration of a doctor with a child holding a teddy bear next to an FDA Rare Disease Priority Review Voucher, with DNA helix and medication imagery https://www.trialfriend.com/blog/hunter-syndrome-avlayah-fda-approval-what-to-do-next https://www.trialfriend.com/blog/hunter-syndrome-avlayah-fda-approval-what-to-do-next Jason Hunter Wed, 08 Apr 2026 12:00:00 GMT 2026-09-25 Avlayah is the first Hunter syndrome therapy in 20 years that can reach the brain. For families who've been waiting, the question isn't whether this matters. The question is what to do about it. News Hunter Syndrome MPS II FDA Approval Rare Disease Medical professional holding an orange card that reads Hunter Syndrome https://www.trialfriend.com/blog/traveling-far-for-clinical-trials https://www.trialfriend.com/blog/traveling-far-for-clinical-trials Jason Hunter Tue, 07 Apr 2026 12:00:00 GMT The trial site is 4 hours away. Or a flight away. Your kid still has school. Your boss needs 2 weeks notice for time off. Nobody warned you about this part. Guide Clinical Trials Travel Patient Access Caregivers Airplane taking off against a sunset sky, representing the long-distance travel many clinical trial patients face https://www.trialfriend.com/blog/dravet-syndrome-treatments-2026-gene-therapy-seizure-management https://www.trialfriend.com/blog/dravet-syndrome-treatments-2026-gene-therapy-seizure-management Jason Hunter Mon, 06 Apr 2026 12:00:00 GMT 2026-09-29 Two disease-modifying therapies for Dravet syndrome now have FDA Breakthrough Therapy designation. For the first time, caregivers have options beyond seizure management. Here is where the science stands right now. Gene Therapy Dravet Syndrome Clinical Trials Rare Disease Epilepsy Medical chart with Dravet syndrome diagnosis and stethoscope https://www.trialfriend.com/blog/bag3-dilated-cardiomyopathy-gene-therapy-affinia-upbeat-trial https://www.trialfriend.com/blog/bag3-dilated-cardiomyopathy-gene-therapy-affinia-upbeat-trial Jason Hunter Mon, 06 Apr 2026 12:00:00 GMT Affinia Therapeutics just received approval to begin dosing patients in the UPBEAT trial, a first-in-human gene therapy study for BAG3-associated dilated cardiomyopathy. A second company, Rocket Pharmaceuticals, is close behind. For the roughly 70,000 patients living with this genetic form of heart failure, targeted treatment has never existed until now. Gene Therapy Dilated Cardiomyopathy Clinical Trials Rare Disease Cardiology BAG3 gene therapy for dilated cardiomyopathy https://www.trialfriend.com/blog/tepezza-subcutaneous-thyroid-eye-disease-obi-trial https://www.trialfriend.com/blog/tepezza-subcutaneous-thyroid-eye-disease-obi-trial Jason Hunter Mon, 06 Apr 2026 12:00:00 GMT Amgen's Phase 3 trial showed that a subcutaneous version of Tepezza delivered via on-body injector matched the efficacy of the IV infusion, with a 77% proptosis response rate. For the roughly 25,000 patients managing TED in the U.S., this could replace 8 hospital infusions with 12 quick injections closer to home. News Clinical Trials Rare Disease Thyroid Eye Disease Amgen Close-up of eyes illuminated by warm light, representing thyroid eye disease https://www.trialfriend.com/blog/als-pridopidine-prevails-phase-3-what-patients-should-know https://www.trialfriend.com/blog/als-pridopidine-prevails-phase-3-what-patients-should-know Jason Hunter Sun, 05 Apr 2026 12:00:00 GMT 2026-09-29 The PREVAiLS study just enrolled its first patient in a Phase 3 trial of pridopidine for ALS. The drug did not meet its primary endpoint in Phase 2, but a specific group of patients showed real signals. Here is what changed, why it matters, and what patients should know. News ALS Clinical Trials FDA Prilenia ALS amyotrophic lateral sclerosis letter blocks with stethoscope representing pridopidine Prevails Phase 3 clinical trial https://www.trialfriend.com/blog/rare-disease-coalition-letter-fda-regulatory-crisis https://www.trialfriend.com/blog/rare-disease-coalition-letter-fda-regulatory-crisis Jason Hunter Wed, 01 Apr 2026 12:00:00 GMT A coalition of nearly 100 patient groups, biotech executives, and investors sent a letter to the Trump administration urging regulatory clarity at the FDA. The numbers behind it tell a story that goes well beyond Wall Street. News FDA Rare Disease Policy Investment Blue-tinted collage of U.S. currency representing FDA funding and rare disease investment https://www.trialfriend.com/blog/igan-treatment-explosion-fabhalta-nejm-biohaven https://www.trialfriend.com/blog/igan-treatment-explosion-fabhalta-nejm-biohaven Jason Hunter Tue, 31 Mar 2026 12:00:00 GMT 2026-09-25 Novartis just published 2-year Fabhalta data in the New England Journal of Medicine showing a 49.3% slowing of kidney function decline. Meanwhile, Biohaven's early-stage degrader program is producing results that could redefine how the disease is treated altogether. 3 years ago, IgAN patients had zero approved therapies. News IgA Nephropathy FDA Novartis Biohaven Complement Human kidney anatomy illustration representing IgA nephropathy treatment breakthroughs with Fabhalta and Filspari https://www.trialfriend.com/blog/gene-therapy-pricing-access-rare-disease https://www.trialfriend.com/blog/gene-therapy-pricing-access-rare-disease Jason Hunter Sun, 29 Mar 2026 12:00:00 GMT 2026-09-24 US gene therapies now list at up to $4.25 million, and 2 have left the US market for lack of buyers. Families almost never pay the list price, though. The 2026 price list, what insurance and Medicaid cover, and the support programs that handle travel and paperwork. Guide Gene Therapy Healthcare Access Rare Disease Policy DNA helix on a dollar bill, symbolizing the cost of gene therapy https://www.trialfriend.com/blog/clinical-trial-ends-continued-access-options https://www.trialfriend.com/blog/clinical-trial-ends-continued-access-options Jason Hunter Wed, 25 Mar 2026 12:00:00 GMT When a clinical trial ends, the drug you have been taking may not yet be available through prescription. Several pathways exist to bridge the gap between trial completion and FDA approval - each with different eligibility, timelines, and requirements. Understanding these options matters. Guide Clinical Trials FDA Patient Access Runner passing a baton on a track, symbolizing the handoff after a clinical trial ends https://www.trialfriend.com/blog/fenebrutinib-ms-phase-3-what-patients-should-know https://www.trialfriend.com/blog/fenebrutinib-ms-phase-3-what-patients-should-know Jason Hunter Wed, 04 Mar 2026 12:00:00 GMT Roche's fenebrutinib hit its primary endpoint in all 3 Phase III trials, showing a 51-59% reduction in relapses for relapsing MS and the first new mechanism to slow disability in progressive MS in over a decade. Here is what that actually means if you or someone you know is living with MS. News Multiple Sclerosis Roche FDA BTK Inhibitor Clinical Trials Brain and nervous system illustration for fenebrutinib BTK inhibitor Phase 3 clinical trial results in multiple sclerosis https://www.trialfriend.com/blog/fda-plausible-mechanism-ultra-rare-disease https://www.trialfriend.com/blog/fda-plausible-mechanism-ultra-rare-disease Jason Hunter Mon, 23 Feb 2026 12:00:00 GMT The FDA announced a new framework that allows individualized therapies for ultra-rare genetic conditions to be approved based on biological plausibility rather than traditional large-scale trials. The policy was partly inspired by a baby with CPS1 deficiency who received a custom CRISPR therapy. News FDA Gene Therapy CRISPR Rare Disease Policy Happy baby playing with colorful toys, representing hope for children with ultra-rare diseases https://www.trialfriend.com/blog/placebos-randomization-clinical-trials-explained https://www.trialfriend.com/blog/placebos-randomization-clinical-trials-explained Jason Hunter Fri, 20 Feb 2026 12:00:00 GMT If you have been offered a spot in a clinical trial, you have probably heard the words placebo and randomization. These concepts can sound intimidating, but they are straightforward once you understand what they actually mean for your experience as a participant. Guide Clinical Trials Patient Education Colored pills and capsules representing placebos and randomization in rare disease clinical trials https://www.trialfriend.com/blog/rett-syndrome-gene-therapy-trials-2026 https://www.trialfriend.com/blog/rett-syndrome-gene-therapy-trials-2026 Jason Hunter Wed, 18 Feb 2026 12:00:00 GMT 2026-09-25 Two gene therapy programs for Rett syndrome now have FDA Breakthrough Therapy designation and are in registrational or pivotal trials. For families weighing whether to pursue a trial, here is what the landscape actually looks like right now. News Gene Therapy Rett Syndrome Clinical Trials Rare Disease FDA Rett Syndrome medical file with stethoscope https://www.trialfriend.com/blog/hidden-costs-of-clinical-trials https://www.trialfriend.com/blog/hidden-costs-of-clinical-trials Jason Hunter Sun, 15 Feb 2026 12:00:00 GMT Clinical trials cover the cost of the investigational treatment, but that is only part of the picture. Travel, lodging, lost wages, and childcare can add up fast, especially for rare disease patients who often have to travel hundreds of miles to reach a trial site. Guide Clinical Trials Patient Access Insurance Iceberg with 'Hidden Costs' text, symbolizing unseen expenses of clinical trial participation https://www.trialfriend.com/blog/regenxbio-hunter-syndrome-gene-therapy-crl https://www.trialfriend.com/blog/regenxbio-hunter-syndrome-gene-therapy-crl Jason Hunter Mon, 09 Feb 2026 12:00:00 GMT 2026-09-29 The FDA issued a complete response letter for RGX-121, a gene therapy designed to treat the neurological damage caused by Hunter syndrome. For the roughly 500 families in the U.S. living with MPS II, this was a major setback in a years-long wait for a treatment that could reach the brain. News Gene Therapy FDA Hunter Syndrome 3D illustration of an AAV viral vector delivering DNA for gene therapy https://www.trialfriend.com/blog/biohaven-sca-fda-what-it-means-for-patients https://www.trialfriend.com/blog/biohaven-sca-fda-what-it-means-for-patients Jason Hunter Sun, 18 Jan 2026 12:00:00 GMT Biohaven's SCA program hit a regulatory wall despite 8 years of FDA collaboration and data showing 70% slowing of disease progression. For patients losing coordination, speech, and independence with no approved treatments, the stakes could not be higher. News FDA Spinocerebellar Ataxia Biohaven Biohaven logo