According to the US Drug and Food Administration (FDA), the approval of a drug means that data on the drug’s effects have been reviewed and the drug is determined to provide benefits that outweigh its known and potential risks for the intended population. In some cases, the approval of a new drug is expedited. Accelerated Approval can be applied to promising therapies that treat a serious or life-threatening condition and provide therapeutic benefit over available therapies.
Approved Neuromuscular Therapies:
All information on this page was taken the US Drug and Food Administration’s website.
SAREPTA THERAPEUTICS
EXONDYS 51
A Treatment for Duchenne muscular dystrophy
Sarepta THERAPEUTIcs
VYONDYS 53
A Treatment for Duchenne Muscular Dystrophy
PTC Therapeutics
Emflaza
A Treatment for Duchenne Muscular Dystrophy (DMD)
Biogen
Spinraza
A Treatment for Spinal Muscular Atrophy (SMA)
Genentech
Risdiplam
A Treatment for Spinal Muscular Atrophy (SMA)
Avexis
Zolgensma
A Treatment for Spinal Muscular Atrophy (SMA)
Sanofi Genzyme
Lumizyme
A Treatment for Pompe disease (acid α-glucosidase (GAA) deficiency)