rare disease

First Opinion By Matthew Might

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

In the Lab By Megan Molteni

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

Politics By Lev Facher

Billions in new research funding sparks a lobbying frenzy among patient advocates

Health Tech By Katie Palmer

With a nudge from AI, ketamine emerges as a potential rare disease treatment

Health By Eric Boodman

With fewer than 400 progeria patients worldwide, testing a CRISPR cure will be challenging

Pharmalot By Ed Silverman

FDA extends shelf life for glaucoma drug discontinued by Pfizer, but patients remain concerned

First Opinion By Emil D. Kakkis

Aduhelm’s accelerated approval offers a promising roadmap for rare neurological diseases

Biotech By Megan Molteni and Nicholas St. Fleur

STAT+ Conversations: A conversation about in vivo gene editing to treat rare disease

Pharmalot By Ed Silverman

In an about-face, Health Canada allows a controversial rare disease drug back on the market

Health By Kevin Lin

Why two scientific powerhouses are teaming up to tackle rare cancers long overlooked in research

First Opinion By Anne E.P. Frosch, Aileen Ahiskali and Chandy C. John

Unaffordable treatment for severe malaria highlights U.S. drug market pitfalls

Health By Erika Check Hayden

The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price

The Readout LOUD By Damian Garde, Meg Tirrell and Adam Feuerstein

Listen: Scott Gottlieb on Covid and grilling, plus how Zolgensma has changed SMA

Pharmalot By Ed Silverman

‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access

Health By Andrew Joseph

Huntington’s community grieves not just one therapeutic setback, but two

Biotech By Adam Feuerstein

In restarted study, Solid Bio reports no serious toxicity in Duchenne patient given ‘safer’ gene therapy

Biotech By Adam Feuerstein

Sarepta wins new drug approval for patients with Duchenne muscular dystrophy, its third marketed treatment

First Opinion By Sanath Kumar Ramesh, Max G. Bronstein and R. Jude Samulski

Ultra-rare but not forgotten: New drug development paradigms to treat the rarest of diseases

Biotech By Elizabeth Cooney

Novartis and the Gates Foundation aim to create a more practical gene therapy for sickle cell patients around the world

Biotech By Matthew Herper

Moncef Slaoui, departing Warp Speed, raises $250 million to test his R&D philosophy in a new company

First Opinion By William S. Smith

The Biden administration needs to look beyond ICER for evaluating drug therapies

In the Lab By Sharon Begley

In a stem cell experiment that seemingly failed, scientists see a possible way to prevent a devastating disease

Health By Anna Goshua

‘Frustrated and panicking’: For some rare disease patients, shortages of protective gear pose a continued threat

Biotech By Damian Garde

Ovid’s treatment for a rare, untreatable disease fails in a key trial

Pharmalot By Ed Silverman

House passes a bill to close a loophole in a law that gives drug makers ‘unfair’ monopolies