Duchenne muscular dystrophy
FDA Approves Duvyzat for DMD
The FDA approved Duvyzat, the first nonsteroidal drug to treat patients with all genetic variants of Duchenne ...
MARCH 22, 2024
Despite Some Setbacks, Strong Specialty Pipeline Seen in 2024
2023 was a record year for new drug approvals, and while 2024 will probably not exceed that record, specialty ...
FEBRUARY 15, 2024
A Record Pace Seen for 2023 Specialty Approvals
The FDA appears to be making up for lost time after the COVID-19 pandemic. The agency is on schedule to approve 51 ...
DECEMBER 4, 2023
FDA Approves Agamree to Treat Duchenne Muscular Dystrophy
The FDA approved Agamree oral suspension for the treatment of Duchenne muscular dystrophy in children aged 24 ...
OCTOBER 27, 2023
More Data Needed Before Ataluren Can Be Recommended
In a 10-to-1 vote, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee said more data ...
SEPTEMBER 28, 2017
FDA Approves Drug to Treat Duchenne Muscular Dystrophy
The FDA today approved the orphan drug, Emflaza by Marathon Pharmaceuticals to treat patients age 5 years and ...
FEBRUARY 9, 2017
Advocates Pushing FDA To Review Duchenne Drug
FDA advisory committee delay triggers concern over status of eteplirsen.
FEBRUARY 8, 2016
