Neuromuscular disease research involves a number of local studies as well as national multisite sponsored trials that evaluate treatments for neuromuscular conditions.
Gillette played a key role in the clinical trials and breakthrough research for the development of Spinraza™ (nusinersen) a drug to treat spinal muscular atrophy (SMA). The drug was recently approved by the FDA, making it the first-ever FDA-approved therapy for SMA.
This research includes the following conditions:
Select Ongoing Studies
Led by Stephen Smith, MD:
- Effects of Growth Hormone Therapy on Bone Health and Muscle Function in Boys With Duchenne Muscular Dystrophy.