FDA: Soliris is Approved to Treat Generalized MG in the United States
FDA: Soliris is approved to treat adults with generalized MG.
Author: Amy Madsen
FDA Grants Orphan Drug Designation to Amicus Therapeutics’ Experimental Combination Drug for Pompe Disease
The drug therapy ATB200/AT2221, meant to treat Pompe disease, has received FDA Orphan Drug Designation.
FDA Grants Orphan Drug Designation to Stealth BioTherapeutics’ Experimental Mitochondrial Myopathy Drug Elamipretide
Elamipretide, which treats muscle weakness caused by mitochondrial disease, has received FDA Orphan Drug Designation
Five Questions with Researcher Maya Maor Nof
Maya Maor Nof of Stanford University School of Medicine received the 2017 SSSI-MDA Fellowship Award to study ALS.
FDA Advisory Committee: Data in Support of Translarna ‘Inconclusive’
The FDA yesterday convened a meeting with the PCNS, to review the NDA, for ataluren (brand name Translarna) to treat some forms of DMD. Following a presentation of the data in support of the NDA by PTC Therapeutics the overwhelming majority (10 out of 11) suggested that although it is possible that Translarna may be effective, the data are inconclusive and more work is needed to determine whether, in fact, it is.
LGMD Genetic Testing Program: What You Need to Know
Do you or a loved one live with LGMD? Learn more about MDA’s LGMD genetic testing program and find out how to make an impact on LGMD research.
Update: Advisory Committee Meeting for Translarna
The FDA on Thursday will convene a meeting with the Peripheral and Central Nervous Systems Drugs Advisory Committee (PCNS), to review the new drug application, or NDA, for ataluren (brand name Translarna) DMD.
Five Questions with Researcher Marshall Hogarth
Dr. Marshall Hogarth was awarded an MDA grant to research LGMD2B and Miyoshi myopathy.
Five Questions with FA Researcher Manuela Corti
Manuela Corti of University of Florida was awarded an MDA grant to study gene therapy in FA
Five Questions with Researcher Eduardo Balsa Martinez
Learn about Dr. Eduardo Balsa Martinez’s research to idenitfy therapeutic targets in mitochondrial diseases.
