Rare Disease News and Guides | FDA Decisions and Trials

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Close-up of a woman's hazel eyes looking upward, dark curls blurred around her face.

NewsOctober 2, 2026FDA decision Oct 15

Enspryng Faces an FDA Decision for Thyroid Eye Disease on October 15th With 1 Trial Won and 1 Lost

A gloved hand turns the lenses of a microscope over a glass tissue slide in a lab.

GuideOctober 2, 2026

The NTRK Fusion Test Most Cancer Patients Never Get, and the 3 Pills That Depend on It

Colorized electron micrograph of immune cells: round pink T cells pressed against a green dendritic cell on a black background.

GuideOctober 1, 2026

Kesimpta vs Ocrevus vs Briumvi, and Why the Same B-Cell Drugs Now Run Through 6 Rare Diseases

A person in a gray T-shirt pinches skin at the waist and holds a prefilled injection pen ready to inject at home.

GuideOctober 1, 2026

Hemophilia A Prophylaxis Compared, From Daily Alhemo to Every-Other-Month Qfitlia, With Denecimig Waiting on the FDA

A hand reaches toward an IV pole with an infusion line hanging from it in a hospital room.

GuideSeptember 30, 2026

Myasthenia Gravis Drugs Compared by Antibody Type, How They Are Given and What You Will Pay

A handful of white, yellow and brown pills in the open palm of a person's hand.

NewsSeptember 30, 2026FDA decision Nov 1

Mitapivat Faces a November 1st FDA Decision for Sickle Cell Disease on the Same Kind of Evidence Oxbryta Had

A man in a grey T-shirt holds an injection pen near his stomach, about to give himself a dose at home.

GuideSeptember 29, 2026FDA decision Nov 30

Atacicept vs Povetacicept for IgA Nephropathy Before the FDA Decision on November 30th

A hand takes a pill from a weekly pill organizer on a dark table, next to a blister pack.

DataSeptember 28, 2026

A Third of Rare Disease Trials Can Shut You Out Over a Medication You Already Take

A young man in a grey hoodie stretches his arms on a sunlit forest path before a run.

GuideSeptember 27, 2026

Alpha-1 Antitrypsin Deficiency Treatment Has Meant a Weekly Infusion Since 1987, and a One-Time Gene Edit Is Now Testing That

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.

GuideSeptember 26, 2026

Your Insurer Denied a Rare Disease Drug and Fewer Than 1 in 100 People Appeal, So Here Is the Playbook

A doctor in a white coat talks with an older man with grey hair as they sit together on a couch.

GuideSeptember 25, 2026

ATTR Amyloidosis Treatment in 2026, 5 Approved Drugs Compared on Survival, Dosing and Side Effects

Three older adults at a table, one pointing at a form while another fills it in.

GuideSeptember 25, 2026

Before You Pick a 2027 Medicare Plan, 8 Things to Check If You Take a Rare Disease Drug

A doctor presses a stethoscope to a patient's chest to listen to their breathing.

NewsSeptember 24, 2026FDA decision Oct 30

INO-3107 Could Become the Second Treatment for Recurrent Respiratory Papillomatosis on October 30th

Two doctors hold up a sheet of brain MRI scans against a bright window to review them.

GuideSeptember 24, 2026

What Clinical Trials Are Available for Acromegaly? 17 Are Open, and 3 Have US Sites for Patients

An open palm holding 3 different capsules against a dark background.

NewsSeptember 23, 2026FDA approved Sep 23

Lirafugratinib Is Approved as Lyrfigtu, the First FGFR2-Only Drug for Bile Duct Cancer

Overhead view of a group of runners on a red track, with one runner falling behind the pack.

NewsSeptember 22, 2026FDA approved Sep 30

Camzyos Is the First Drug Approved for Teenagers With Obstructive HCM, Cleared on September 30th

A toddler walking across grass while holding a parent's hand on each side.

NewsSeptember 21, 2026FDA approved Sep 18

Aqneursa Is the First Approved Treatment for Ataxia-Telangiectasia, Nearly 8 Months After the Disease's Biggest Trial Failed

A newborn lying on his stomach and lifting his head to look to the side.

NewsSeptember 18, 2026FDA approved Sep 28

Emcitate Is Approved for MCT8 Deficiency, and the Trials Show It Treats the Body but Not the Brain

A young patient steadying herself on parallel bars while a physical therapist records her motor function assessment on a clipboard.

NewsSeptember 17, 2026FDA approved Sep 11

Isembyld Is Approved for SMA as the First Muscle-Targeted Add-On to Spinraza and Evrysdi

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.

NewsSeptember 17, 2026FDA approved Sep 17

UX111 Is Approved as Fayuvi, the First Treatment for Sanfilippo Syndrome, and the Label Says Exactly Who It Is For

A gloved hand lifting a labeled specimen tube from a rack in a clinical laboratory.

GuideSeptember 16, 2026

6 Approved Drugs for IgA Nephropathy and What Their Labels Actually Promise

A bag of donated blood hanging on a line above a drip chamber during a transfusion.

NewsSeptember 16, 2026

Alpha-Gal Syndrome and Blood Transfusions, What Type O Patients Should Tell Their Hospital

An open hand holding pills next to a weekly pill organizer and a glass of water on a light blue surface.

NewsSeptember 15, 2026

1 Year of Palsonify and What Switching From Acromegaly Injections to a Pill Involves

Green and white capsules sealed in clear blister packs on a teal background.

NewsSeptember 15, 2026

A New AI Tool Called MARRVEL-MCP Is Changing the Slowest Part of Rare Disease Diagnosis

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

GuideMay 20, 2026

Rare Disease Diagnoses That Started With Something Else Entirely

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.

GuideMay 12, 2026

Lone Star Tick Bites and the Rare Diseases They Cause from Alpha-Gal Syndrome to Heartland Virus

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

GuideMay 9, 2026

Rystiggo for Myasthenia Gravis and How It Compares to Vyvgart and Imaavy

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

GuideMay 8, 2026

Alpha-Gal Syndrome: Medications, Vaccines, and Medical Products That Can Trigger a Reaction

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

GuideMay 7, 2026

Casgevy vs. Lyfgenia: Comparing Sickle Cell Gene Therapies

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

GuideMay 6, 2026

School Accommodations for a Child with a Rare Disease: A Parent's Field Guide

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

GuideMay 2, 2026

hEDS, MCAS, POTS, MTHFR, and Tick-Borne Coinfections: When Rare Diagnoses Stack

Empty hospital procedure room with surgical light, IV pole, and adjustable bed, representing the inpatient setting where heparin-induced thrombocytopenia develops and is treated

NewsApril 30, 2026

Heparin-Induced Thrombocytopenia: A New Drug Path After 25 Years

Solitary figure on a rocky outcrop overlooking layered mountain ridges in golden morning light, representing the first regulatory crossing for in vivo gene editing

NewsApril 30, 2026FDA decision Mar 10, 2027

Lonvo-Z and the First Phase 3 Win for In Vivo Gene Editing in HAE

Aerial drone view of a long winding road cutting through a dense pine forest, representing the years-long search for a rare disease diagnosis

GuideApril 29, 2026

Why Rare Disease Diagnoses Still Take 6 Years, and How Genome Sequencing Can Cut It to Weeks

DNA double helix representing gene editing therapies for rare diseases under the FDA plausible mechanism framework

NewsApril 24, 2026

The FDA's Plausible Mechanism Framework Could Unlock Gene Therapies for the Rarest Diseases

Hospital ward representing emergency room challenges for rare disease patients like sickle cell and Addison's disease

GuideApril 21, 2026

What Happens When You Take a Rare Disease to the ER

Detailed illustration of a blacklegged tick, the primary vector for Lyme disease, babesiosis, and anaplasmosis

GuideApril 19, 2026

Ticks, Rare Disease, and the Coinfections Lyme Patients Have Been Told Don't Exist

Cipher wheel representing how to decode and read a ClinicalTrials.gov listing for rare disease clinical trials

GuideApril 17, 2026

How to Read a ClinicalTrials.gov Listing Without a Medical Degree

Scientist using forceps in a clinical research laboratory setting

GuideApril 16, 2026

A Patient's Guide to Clinical Trial Participation

PerspectiveApril 15, 2026

Most Clinical Trial Finders Are Recruitment Tools in Disguise. We Built Something Else.

Travere Therapeutics Filspari sparsentan FDA approval for FSGS focal segmental glomerulosclerosis rare kidney disease

NewsApril 14, 2026

Filspari Is the First FDA-Approved Treatment for FSGS. Here's What That Actually Means for Patients.

Empty clinic chairs representing the rare disease clinical trial enrollment slowdown despite record FDA orphan drug approvals

NewsApril 13, 2026

A Quiet Crisis in Rare Disease Research: Trial Enrollment Is Slowing Just as Treatments Start Working

Person in a suit holding a torn piece of paper that reads Are You Covered, representing insurance coverage questions for rare disease patients

GuideApril 11, 2026

Insurance Denied Your Medication. You Have More Power Than They Want You to Know.

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

GuideApril 9, 2026

That Drug Your Child Takes? A Voucher Worth Millions Helped Make It Happen.

Medical professional holding an orange card that reads Hunter Syndrome

NewsApril 8, 2026

Avlayah Is the First New Hunter Syndrome Treatment in 20 Years. Here's What Families Should Do Next.

Airplane taking off against a sunset sky, representing the long-distance travel many clinical trial patients face

GuideApril 7, 2026

You Got Into a Clinical Trial. Now You Have to Get There.

Medical chart with Dravet syndrome diagnosis and stethoscope

Gene TherapyApril 6, 2026

Dravet Syndrome Treatments in 2026: From Seizure Management to Gene Therapy

BAG3 gene therapy for dilated cardiomyopathy

Gene TherapyApril 6, 2026

First Gene Therapy for BAG3 Heart Failure Enters Clinical Trials: What Patients Should Know

Close-up of eyes illuminated by warm light, representing thyroid eye disease

NewsApril 6, 2026

Tepezza Just Got a Major Upgrade: What the New At-Home Injection Means for Thyroid Eye Disease Patients

ALS amyotrophic lateral sclerosis letter blocks with stethoscope representing pridopidine Prevails Phase 3 clinical trial

NewsApril 5, 2026

A New ALS Trial Is Recruiting and It Learned from What Came Before

Blue-tinted collage of U.S. currency representing FDA funding and rare disease investment

NewsApril 1, 2026

84% of Investors Are Pulling Back from Rare Disease. Here Is Why That Should Alarm Every Patient.

Human kidney anatomy illustration representing IgA nephropathy treatment breakthroughs with Fabhalta and Filspari

NewsMarch 31, 2026

From Zero Treatments to 5 in 3 Years: The IgA Nephropathy Breakthrough Nobody Saw Coming

DNA helix on a dollar bill, symbolizing the cost of gene therapy

GuideMarch 29, 2026

What a $4 Million Gene Therapy Actually Costs a Family in 2026

Runner passing a baton on a track, symbolizing the handoff after a clinical trial ends

GuideMarch 25, 2026

What Happens After a Clinical Trial Ends - Your Options for Continued Access

Brain and nervous system illustration for fenebrutinib BTK inhibitor Phase 3 clinical trial results in multiple sclerosis

NewsMarch 4, 2026

A New Kind of MS Drug Just Cleared Its Biggest Hurdle: What Fenebrutinib Means for Patients

Happy baby playing with colorful toys, representing hope for children with ultra-rare diseases

NewsFebruary 23, 2026

One Baby Changed Everything: How the FDA Opened the Door for Ultra-Rare Disease Treatments

Colored pills and capsules representing placebos and randomization in rare disease clinical trials

GuideFebruary 20, 2026

What Placebos and Randomization Actually Mean for You (In Plain English)

Rett Syndrome medical file with stethoscope

NewsFebruary 18, 2026

MECP2 Gene Therapy for Rett Syndrome: What Families Should Know About the Trials in 2026

Iceberg with 'Hidden Costs' text, symbolizing unseen expenses of clinical trial participation

GuideFebruary 15, 2026

The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them)

3D illustration of an AAV viral vector delivering DNA for gene therapy

NewsFebruary 9, 2026

Regenxbio's Hunter Syndrome Gene Therapy Rejected: What Happened and What It Means

NewsJanuary 18, 2026

When the Measuring Stick Moves: Biohaven, the FDA, and What It Means for SCA Patients

Questions about Trial Friend News and Guides

What does Trial Friend News and Guides cover?

Rare disease clinical trial results, FDA approval decisions, gene therapy access, drug pipelines, insurance and access problems, and treatment guides, written in plain language for patients and caregivers rather than for clinicians.

Where does the information come from?

Every post is built from primary sources: FDA announcements and drug labels, ClinicalTrials.gov records, company announcements, and peer-reviewed studies. Each post lists its sources at the end. No pharmaceutical company sponsors or influences what we publish.

Who writes Trial Friend News and Guides?

Jason Hunter, the independent writer and editor behind Trial Friend. No drug company funds or reviews his work. He uses AI tools to help research, draft and check posts, and approves every post against its cited sources before it is published. He is not a clinician, and nothing here is medical advice.

How do you keep posts current after an FDA decision?

Posts written ahead of an FDA decision are updated in place the day the outcome is announced, and the date of the change is shown next to the publish date. The FDA calendar on Trial Friend carries each outcome with its source.

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