rare disease

Biotech Adam Feuerstein

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

In the Lab Megan Molteni

STAT Plus: New CRISPR tools could fix diseases caused by large DNA rearrangements, scientists report

Politics Nicholas Florko

STAT Plus: 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

STAT Plus: 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

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

Politics Lev Facher

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

Health Tech Katie Palmer

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

Politics Nicholas Florko

STAT Plus: Congress presses the FDA to move faster on ALS drugs

First Opinion Guadalupe Hayes-Mota

21 states give rare disease patients a seat at the table. The other 29 need to follow suit

Health Eric Boodman

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

In the Lab Megan Molteni

Landmark gene therapy trial points to a wider window to alter course of rare disease

Pharmalot Ed Silverman

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

First Opinion Emil D. Kakkis

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

Biotech Megan Molteni and Nicholas St. Fleur

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

Pharmalot Ed Silverman

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

First Opinion Calaneet Balas

FDA should lead the way on new ALS treatments, not Canada and Europe

Health Kevin Lin

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

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

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

Pharmalot Ed Silverman

STAT Plus: After repeated losses in the U.S., Catalyst wins a victory in Canada over a rare disease drug

Health Erika Check Hayden

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

The Readout LOUD Damian Garde, Meg Tirrell and Adam Feuerstein

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

Pharmalot Ed Silverman

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

Health Andrew Joseph

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

Biotech Adam Feuerstein

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

Biotech Adam Feuerstein

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

First Opinion 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