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
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
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
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