MUSCULAR DYSTROPHIES

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease by allowing the body to produce a shorter but still functional dyst…

This study tests an oral drug called SAT-3247 in about 51 boys aged 7 to 10 with Duchenne muscular dystrophy (DMD). The goal is to find the best dose and see if it is safe and helps muscle strength. Participants will take the drug or a placebo for 12 weeks, and all will continue …

This early study tests a brain implant in 5 adults with severe paralysis (from spinal cord injury, ALS, or similar conditions). The goal is to see if the device is safe and can help users control assistive devices like computers or wheelchairs. The implant records brain signals a…

This study is testing a device called the mScan that uses a tiny, painless electrical current to measure muscle health in about 6 seconds. Researchers want to see if it can give similar information as an MRI, but faster and more conveniently. The study involves 150 adults with an…

This early-stage study tests the safety of a brain implant (BrainGate system) in up to 3 people with severe paralysis or locked-in syndrome. The goal is to see if participants can use their thoughts to control a computer cursor or other assistive devices. The study focuses on saf…

This study aims to find better ways to measure fatigue and walking problems in people with neuromuscular diseases like muscular dystrophy and spinal muscular atrophy. Researchers will track walking speed during a 6-minute test and use a wearable sensor to monitor daily activity f…

This early-stage study tests whether a brain implant can help people with severe paralysis from conditions like ALS, spinal cord injury, or stroke control devices using their thoughts. The implant records brain signals to decode text or synthesized speech. Only 3 adults will part…

This study tests a brain implant that reads signals from the brain to help people who have lost the ability to speak fluently communicate by simply trying to talk. Two adults with conditions like spinal cord injury or ALS will be enrolled. The goal is to understand how speech is …