Zero ad trackers.We never sell your data.

Rare Disease News and Guides

FDA decisions, clinical trials and treatments, explained in plain language for patients and caregivers.

Next FDA decisions

Full calendar →
A hand in a gray patterned mitten holds a covered hot drink cup in a cold, misty forest.
Guide

Cold Agglutinin Disease Treatment Before Winter, and Why Enjaymo and Rituximab Help Different Symptoms

Cold agglutinin disease causes 2 separate problems, red blood cell destruction and cold-triggered blue, numb fingers, and its treatments do not fix both the same way. What the research shows about winter, what a hospital needs to know, and how Enjaymo, rituximab and rituximab with bendamustine compare.

An older man in a tan jacket reaches the top of a long stone staircase, head down, with a pine tree behind him.
GuideFDA decision Nov 14th

Starting Obstructive HCM Treatment With Myqorzo or a Beta Blocker, and What the MAPLE-HCM Trial Found

A man presses a pen injector into the front of his thigh while seated at home in a white T-shirt.
Comparison

Redemplo vs Tryngolza for Familial Chylomicronemia Syndrome, Compared on Injections, Side Effects, Price and the Gene Test Insurers Ask For

A woman with short curly hair holds a clear nebulizer mask over her nose and mouth.
FDA DecisionDecision moved to Nov 22nd

What Patients With Autoimmune PAP Told the FDA, and How Molbreevi Measured Up Before Its November 22nd Decision

All articles

A hand holds a digital blood pressure monitor while a grey cuff is wrapped around the upper arm.
FDA Decision

Cushing's Syndrome Could Get a New Cortisol-Blocking Pill on December 17th, After the FDA Questioned Its Trial and Liver Safety

Rows of blood collection tubes with purple, gold, teal and tan caps standing in a laboratory rack.
Guide

Lyme and Coinfection Tests, Which Test Finds What, Who Decides What Counts as Positive, and Why Most Visits Offer Only One

Close-up of tree growth rings fanning out from the center of a freshly cut log, in cream and deep red.
Guide

Adults With SMA Now Have a One-Time Gene Therapy in Itvisma, and Insurers Do Not All Agree on Who Gets It

An older hand holds a metal colander up to the sun, casting a shadow of tiny holes onto a white board.
FDA Decision

Gazyva for Membranous Nephropathy, What the 37% Remission Result Means for Your Next Treatment

A man walking away down a tree-lined sidewalk with a wooden cane on a sunny day.
News

Troriluzole Is Back Under FDA Review for Spinocerebellar Ataxia, and This Time SCA Families May Get a Say

A mother types at a laptop on her dining table while her young son draws on the couch behind her.
Guide

Is Your Rare Disease on the FDA's Listening List? Many Are Not, and Comments on the New Rare Disease Framework Close December 11th

An older couple holding hands as they walk across a sunny town square.
GuideFDA approved Aug 24th

Who Can Get Imaavy, the First FDA-Approved Treatment for Warm Autoimmune Hemolytic Anemia

Gloved hands pipetting a sample into a tube rack in a lab, the first step of the gene tests run at an AML diagnosis.
Guide

Your AML Gene Results May Come Back After Chemo Starts, and They Decide Which of 8 Targeted Drugs You Can Get

A person in jeans climbing an outdoor staircase with one hand on the metal railing, a brick wall behind.
News

A Natural Sugar Could Become the First Treatment for Limb-Girdle Muscular Dystrophy 2I/R9 on November 27th

A hand reaching into a weekly pill organizer to take the day's medicine.
Guide

Iqirvo or Livdelzi? Choosing the Next PBC Pill When Ursodiol Falls Short

A clear IV infusion line in soft focus against a bright window, the way Gaucher enzyme infusions are given.
GuideFDA decision Nov 25th

What a Year of Gaucher Disease Treatment Costs, From Cerezyme Infusions to the Venglustat Pill Due for an FDA Decision November 25th

A young man in a wheelchair at sunset, seen from behind with one hand on the wheel rim.
FDA DecisionDecision moved to Nov 22nd

Deramiocel Now Asks the FDA to Protect Arm Function in Duchenne, With a Decision Due November 22nd

A researcher in a white coat working at a bench in a hospital laboratory.
Guide

Who Runs Your Rare Disease's Clinical Trials, and Why It Changes What You'll Be Offered

A baby in a car seat wraps a small hand around an adult's thumb, photographed in black and white.
GuideDecision moved to Nov 17th

What Can a Baby With Niemann-Pick Type C Be Treated With Before the November 17th Adrabetadex Decision?

A weathered U.S. Mail mailbox with its red flag down, mounted beside a wooden fence post.
Guide

Mailed Prescriptions From Canada After January 20th and the FDA Path Left for Domperidone

A woman rests her chin in her hand as she looks at her phone at a table in a dark room.
Guide

Is a Health Website Tracking You? 5 Free Checks Before You Type a Diagnosis

A smiling woman reads a printed letter at her kitchen table next to a bowl of apples and a blue mug.
Guide

What Your Wilson Disease Copper Test Results Mean on Penicillamine, Trientine or Zinc

A pharmacist in a white coat reaches for a medicine box on a shelf in a pharmacy stockroom.
Guide

Generic Promacta Keeps the Same ITP Dose While Alvaiz Changes the Number

Two adults stand side by side holding hands, one in a blue shirt and one in a patterned shirt.
GuideFDA decision May 1, 2027

Ingrezza, Austedo or Tetrabenazine for Huntington's Chorea, and Where the AMT-130 Gene Therapy Stands Now

A parent rests a hand on a toddler's head as the little boy looks up at the camera.
GuideFDA decision Feb 28, 2027

Achondroplasia Treatment for Your Child, Voxzogo, Yuviwel and the 2 FDA Decisions Coming in February

Close-up of an older woman placing a white pill between her lips.
News

Admilparant, Rebuilt After a Liver Problem, Faces Liver Questions in Its Phase 3 IPF Trials

Hands holding open an old family photo album of black-and-white pictures, for a guide to TTR gene testing and hereditary ATTR amyloidosis across generations.
Guide

Diane Wilkie's TTR Gene Test Came Back Positive Before She Had a Single Symptom

Three empty red chairs in a clinic waiting room beside a sunlit window, for families waiting on 10 rare disease FDA decisions in November 2026.
News

7 Rare Disease FDA Decisions Fall in the Last 9 Days of November

A young boy climbs a wooden staircase on his hands and feet while his sister watches from below.
Analysis

Duchenne Exon Skipping by Exon, Which Drug Matches Your Child and 2 New Ones Under FDA Review

Close-up of a woman's hazel eyes looking upward, dark curls blurred around her face.
NewsFDA decision Jan 10, 2027

Enspryng Is Approved for Thyroid Eye Disease as the First At-Home Shot, With 1 Trial Won and 1 Lost

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

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.
Guide

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.
Guide

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.
Guide

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.
NewsDecision moved to Feb 1, 2027

Mitapivat Faces a February 1st FDA Decision for Sickle Cell Disease, With a Safety Program Question and the Oxbryta History in View

A man in a grey T-shirt holds an injection pen near his stomach, about to give himself a dose at home.
GuideFDA decision Nov 30th

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.
Data

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.
Guide

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.
Guide

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.
Guide

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.
Guide

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.
NewsFDA approved Oct 30th

Voceryq (INO-3107) Approved as the Second Treatment for Recurrent Respiratory Papillomatosis

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

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.
NewsFDA approved Sep 23rd

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

Overhead view of a group of runners on a red track, with one runner falling behind the pack.
NewsFDA approved Sep 30th

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.
NewsFDA approved Sep 18th

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

A newborn lying on his stomach and lifting his head to look to the side.
NewsFDA approved Sep 28th

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.
NewsFDA approved Sep 11th

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.
NewsFDA approved Sep 17th

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.
Guide

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.
News

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.
News

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.
News

We Built a Free Alpha-Gal Medication Checker

Calendar grid with weeks of days filled in teal, counting toward one day marked in amber.
Guide

The FDA Approved a Drug for Your Rare Disease. When Do You Actually Get It?

Two people holding hands in comfort, one resting a hand on the other's.
News

When a Clinical Trial Fails and It Was Your Family's Trial

Empty two-lane desert highway running straight toward distant mountains.
Data

The Median Rare Disease Trial Recruits at Exactly One US Site

FDA headquarters sign in front of the agency's White Oak campus in Maryland.
Guide

What an FDA Clinical Hold Means When You're the One in the Trial

X-ray of a human torso and shoulders, the kind of imaging used to track new bone growth in FOP.
NewsFDA approved Sep 25th

Zilurgisertib Could Give FOP Patients a Third Treatment on September 26th

Fluorescence microscope view of cells glowing purple, blue, and green against a black background.
Data

The State of Rare Disease Clinical Trials With 75% of 2026 Behind Us

A hand holds an hourglass with sand running through it, marking the final weeks before 8 rare disease FDA decisions in September 2026.
News

4 Rare Diseases Could Get Their First Treatment This September

Chemical structure of ezogabine, the Kv7-opening seizure drug withdrawn from the market in 2017.
News

Opakalim, Azetukalner, and the Race to Bring Back Epilepsy's Lost Off Switch

Stylized DNA helix glowing across a dark background, representing AI-assisted rare disease variant interpretation.
News

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
Guide

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.
Guide

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
GuideFDA approved Aug 24th

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

A vial of Adrenalin (epinephrine injection, USP, 1mg/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
Guide

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
Guide

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
Guide

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
Guide

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
News

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
NewsFDA 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
Guide

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
News

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
Guide

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
Guide

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
Guide

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

Scientist using forceps in a clinical research laboratory setting
Guide

A Patient's Guide to Clinical Trial Participation

Perspective

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
News

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
News

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
Guide

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
Guide

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

Medical professional holding an orange card that reads Hunter Syndrome
News

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
Guide

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

Medical chart with Dravet syndrome diagnosis and stethoscope
Gene Therapy

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

BAG3 gene therapy for dilated cardiomyopathy
Gene Therapy

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
News

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
News

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
News

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

Illustration of human kidney anatomy
NewsFDA approved Jul 16th

IgA Nephropathy Went From Zero Approved Drugs to 6 in Under 5 Years, and Where Fabhalta Fits

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

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
Guide

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
News

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
News

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
Guide

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

Rett Syndrome medical file with stethoscope
News

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
Guide

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
News

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

Biohaven logo
News

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.

Is Trial Friend News and Guides free?

Yes. Every article, drug guide, and clinical trial analysis on Trial Friend is free, with no account, subscription, or login required.

Use a feed reader? Every new post is in the Trial Friend news and guides RSS feed.