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
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
Pharmalot By Ed Silverman House passes a bill to close a loophole in a law that gives drug makers ‘unfair’ monopolies
By Damian Garde, Meg Tirrell and Adam Feuerstein Listen: Bracing for a pandemic winter, Biogen’s fortune at the FDA, and remembering a remarkable 12-year-old
First Opinion By Wolfram Nothaft, Gregory Moore and Yann Le Cam Using telehealth to revolutionize the speed of making rare disease diagnoses
Biotech By Elizabeth Cooney Wary hemophilia patients say they’re willing to wait longer for a safe gene therapy
Biotech By Adam Feuerstein and Damian Garde Stunning FDA rejection signals potential new hurdles for gene therapies
Pharmalot By Ed Silverman A long-awaited report on orphan drugs in Europe suggests incentives to pharma need change
In the Lab By Sharon Begley Gene-editing discovery could point the way toward a ‘holy grail’: cures for mitochondrial diseases
First Opinion By Christopher Anselmo I’ve been cared for by dozens of clinicians in nearly 20 years. I’m in awe of their work on Covid-19
Pharmalot By Ed Silverman FDA closes review of Novartis data integrity scandal without punishing the drug maker
Biotech By Damian Garde To quell unpredictable allergic reactions, an experimental drug takes aim at a genetic cause, not symptoms
Pharmalot By Ed Silverman Oh, Canada! Vertex battles another country over access to cystic fibrosis treatments
First Opinion By Ethan J. Weiss Should ‘broken’ genes be fixed? My daughter changed the way I think about that question
Pharmalot By Ed Silverman Pharma execs criticize a drug maker over pricing and claim orphan drug law is being misused
Off the Charts By Jennifer Adaeze Okwerekwu As medical professionals, my husband and I know the Nigerian travel ban is cruel. Now the pain is personal
Health By Andrew Joseph and Ed Silverman A lottery like no other offers up a cutting-edge medicine — with lives on the line
Adam's Take By Adam Feuerstein If biotech had a Mount Rushmore, whose heads would you chisel in the granite?
