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rare diseases

News on rare diseases, including the development of new treatments, the challenges of diagnosis and treatment and the stories of patients and their families. The page features in-depth reporting on genetic diseases, immunodeficiencies, metabolic disorders and more.

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Biotech Jason Mast

STAT Plus: Pfizer kills hemophilia gene therapy deal, imperiling Sangamo

Don't Miss STAT Staff

STAT stories you might have missed in 2024

Health Jason Mast

(Your name here) gene therapy: new fund-raising gambit for family with rare disease

First Opinion Shelby Kinsey

I have a rare form of ALS. I have to fight my insurance company every six months for a game-changing drug

The Readout LOUD Elaine Chen, Allison DeAngelis, and Adam Feuerstein

The pharma industry’s silence on RFK Jr., and efforts by parents to develop gene therapies for their children

Adam's Take Adam Feuerstein

STAT Plus: Why Stoke Therapeutics’ Dravet syndrome drug is worth watching

Biotech Jason Mast

STAT Plus: This father built a gene therapy for his son. Now comes the harder part: saving others’ children, too

Pharma Adam Feuerstein and Jonathan Wosen

STAT Plus: Sanofi advances a drug for a rare, platelet-destroying disease

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Biotech Jason Mast

STAT Plus: Novartis buys gene therapy startup Kate Therapeutics, joining pursuit of muscular dystrophy treatment

Biotech Adam Feuerstein

STAT Plus: Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy

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First Opinion Kara Ayik

My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?

Adam's Take Adam Feuerstein

STAT Plus: Capricor and the FDA: A daring, risky filing strategy

Adam's Take Adam Feuerstein

STAT Plus: Study on Sarepta Therapeutics’ Duchenne therapy diverges from the company line

Biotech Jason Mast and Adam Feuerstein

STAT Plus: Perplexing results from Duchenne muscular dystrophy trial raise questions about gene therapies

First Opinion Peter J. Pitts

STAT Plus: The biggest challenge facing the FDA’s new Rare Disease Innovation Hub

Letters to the editor Torie Bosch

First Opinion readers on Covid and Trump, rare diseases, and institutional neutrality

Morning Rounds Timmy Broderick

Morning Rounds: Hospitals’ IV fluid shortage, Bill Nye, and AI skeptics

Biotech Adam Feuerstein

STAT Plus: Sarepta’s gene therapy is fully approved, but its stock has tanked. Here’s why

Biotech Jason Mast

STAT Plus: 7 children developed blood cancer after Bluebird Bio gene therapy for rare neurological disease

Biotech Jason Mast

STAT Plus: As U.S. efforts stall, China pushes ahead with CRISPR treatments for muscular dystrophy

Opinions+ Emil D. Kakkis

STAT Plus: Traditional randomized trials don’t work for ultra-rare diseases like Barth syndrome

Morning Rounds Theresa Gaffney

The 2024 Medicine Nobel, and remembering Sammy Basso

Exclusive Matthew Herper

STAT Plus: Janet Woodcock, former FDA official, joins board of patient group focused on ‘rediscovering’ old drugs

Biotech Elaine Chen

STAT Plus: Scholar Rock drug for spinal muscular atrophy succeeds in late-stage study

Health Eric Boodman

Sammy Basso, progeria advocate, has died at 28

Health Andrew Joseph

STAT Plus: Dispute over Duchenne gene therapy highlights thorny access issues

Adam's Take Adam Feuerstein

STAT Plus: Why Biohaven will test the FDA’s regulatory limits

Pharma Jason Mast

STAT Plus: FDA approves first drug for rare neuron disease, Niemann-Pick type C

Politics John Wilkerson and Jason Mast

STAT Plus: House panel passes pediatric rare disease bill, setting it up for government funding bill