Sarepta to seek approval for gene therapy in rare muscular dystrophy
An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of…
Coverage of Sarepta Duchenne drug may be halted by N.Y. state Medicaid
In a setback for Sarepta Therapeutics, a New York panel has voted unanimously to recommend the state Medicaid…
Knowing patient’s genetic make-up may change the way medicines are prescribed
SINGAPORE – Not all medicines work the same way on every patient, and part of the answer lies…
Acadia Pharmaceuticals drug for rare genetic condition fails late-stage study
Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…
Advancing rare diseases care
image: ©spawns| iStock Lorna Rothery spoke with Victoria Hedley, co-lead of the Rare Disease Research UK Hub, about…