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rare disease

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First Opinion Craig Martin

There’s no ‘Moonshot’ or ‘Warp Speed’ for rare diseases. There should be

Health Jared Whitlock

A mother, shaped by tragedy, embarks on a mission to advance custom medicines

In the Lab Megan Molteni

Provocative new findings suggest a surprising cause of Down syndrome: cells linked to aging

Special Report Eric Boodman

How medicine erased Black women from a ‘white man’s disease’

Health Usha Lee McFarling

Millions of Americans carry the sickle cell trait, many without knowing it. Could they be at risk for severe Covid-19?

Health Damian Garde

A new treatment promises to make little people taller. Is it an insult to ‘dwarf pride’?

Health Adam Feuerstein and Matthew Herper

‘A game-changer’: How Vertex delivered on cystic fibrosis

First Opinion Nathan Yates

I have spinal muscular atrophy. Critics of the $2 million new gene therapy are missing the point

All Coverage

Exclusive Ed Silverman

Novartis reports two children died from acute liver failure after treatment with Zolgensma gene therapy

A STAT Investigation Allison DeAngelis, Adam Feuerstein and Jason Mast

At pioneering center for gene therapy, Jim Wilson presided over toxic, abusive workplace, staffers say

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First Opinion Irene Shapiro

The Morris ALS Principles: A model for empowering all disease communities

Pharmalot Ed Silverman

‘No magic bullet’: For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges

Biotech Damian Garde

FDA advisers endorse Bluebird gene therapy for rare blood disorder

Biotech Damian Garde

FDA advisers unanimously endorse Bluebird’s gene therapy for rare disorder

First Opinion Darcy Krueger and Emanual Maverakis

The Access to Rare Indications Act could be a game changer for millions of Americans

In the Lab Andrew Joseph

Child’s autopsy from 2008 becomes a clue to mystifying polio-like illness

First Opinion Brian Wallach and Mike Braun

Take a stand for those who can’t with the Promising Pathway Act

First Opinion Walter G. Johnson and Diana M. Bowman

It’s time for Congress to stop blocking mitochondrial replacement therapy

First Opinion Bernard Zipprich

Will the shadow of Aduhelm cloud FDA’s decision-making on an ALS therapy?

Pharmalot Ed Silverman

A $2.1 million price tag for Bluebird Bio’s gene therapy is cost-effective, analysis finds

Video Chat Matthew Herper

Virtual Event: CRISPR Quest

In the Lab Eric Boodman

This family carried a rare mutation that should have been lethal. What was keeping them alive?

Health Jonathan Wosen

Researchers shatter the speed record for diagnosing rare genetic diseases with DNA sequencing

In the Lab Megan Molteni

Last-gasp gene therapy saved a Syrian refugee’s life. Five years later, the boy is thriving and a clinical trial is starting

Biotech Matthew Herper

Acadia drug meets endpoints for treating Rett syndrome, a rare neurological disease

Health Andrew Joseph

Sequencing whole genomes helps diagnose far more rare diseases, study shows

Biotech Adam Feuerstein

Centessa drug shows early promise for inherited lung disease, but liver safety issue seen in small study

Politics Nicholas Florko

Drug makers failed to block House provision cracking down on orphan drug tax credits

First Opinion Emily Milligan and Katherine R. McCurdy

Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs

First Opinion Podcast Patrick Skerrett

Listen: A father describes the legacy of his son’s ultra-rare disease

Biotech Kate Sheridan

Startup Spotlight: Vigil Neuroscience hopes to eventually target the rare disease ALSP

First Opinion Matthew Might

Scientific, moral imperatives underlie including rare disorders in the ARPA-H mandate

In the Lab Megan Molteni

Scientists plumbing mysterious RNAs in the human genome find a possible treatment for an inherited disorder