Insmed Gene Therapy Llc

This is an early safety study testing a single spinal injection of an experimental gene therapy called INS1201. It involves up to 12 young boys, aged 2 to under 5 years old, who have Duchenne muscular dystrophy and can still walk. The main goal is to see if the treatment is safe …

This is an early, small study to find a safe dose of an experimental gene therapy called INS1202 for people with ALS. It will test a single injection into the spinal fluid of about 23 adults with ALS, including some with a specific genetic mutation (SOD1) and some without. The ma…