Commercial Treatments

Superhero

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)