rare disease

First Opinion By Brian Wallach and Mike Braun

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

First Opinion By Walter G. Johnson and Diana M. Bowman

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

First Opinion By Bernard Zipprich

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

Pharmalot By Ed Silverman

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

Video Chat By Matthew Herper

Virtual Event: CRISPR Quest

Health By Jonathan Wosen

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

In the Lab By 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 By Matthew Herper

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

Biotech By Adam Feuerstein

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

Politics By Nicholas Florko

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

First Opinion By 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 By Patrick Skerrett

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

Biotech By Kate Sheridan

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

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

Politics By Nicholas Florko

Congress presses the FDA to move faster on ALS drugs

First Opinion By Guadalupe Hayes-Mota

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

Health By Eric Boodman

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

In the Lab By Megan Molteni

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

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