Insmed Gene Therapy Llc

This early-stage study tests a single injection of a gene therapy called INS1201 in 12 young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to see if the treatment is safe and where it goes in the body. Researchers will also look for sign…

This early-stage study tests a single injection of INS1202, a gene therapy, given into the spinal fluid of people with ALS. The main goal is to check safety and find the right dose. About 23 adults aged 18 to 79 with either a specific SOD1 gene mutation or no known genetic cause …