NEUROMUSCULAR DISEASES (NMD)

This study tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a genetic disease that damages nerves. The goal is to see if the treatment can stop or slow the disease by lowering a harmfu…

This study tests whether a virtual reality walking program at home is feasible and helpful for children aged 6 to 17 with conditions like cerebral palsy, neuromuscular diseases, or obesity. Participants use a special treadmill and VR headset for about 30 minutes, three times a we…

This study tests a special exercise method using whole-body electrical muscle stimulation (WB-EMS) in adults with neuromuscular diseases like ALS, SMA, and muscular dystrophy. Because these conditions make normal exercise hard, WB-EMS directly triggers muscle contractions without…

This study is testing a special fabric shoulder brace that mimics muscle action to help people with neuromuscular disorders (like muscular dystrophy or ALS) move their arms better. About 30 participants will be evaluated with and without the brace to see if it improves reach, str…

This study checks whether a common nerve test called needle EMG is safe for people taking blood thinners (DOACs). About 30 minutes after the test, researchers use ultrasound to look for small muscle bleeds (hematomas) where the needle went in. The goal is to see if the risk of bl…

This study is creating a large collection of blood, skin, and spinal fluid samples along with health information from 2,500 adults who have neuromuscular diseases (like ALS, neuropathy, or muscular dystrophy). The goal is to store these materials and share them with other researc…

This study looks back at the past 10 years to understand how many people in Denmark use a breathing machine at home through a tube in their windpipe. Researchers will track which diseases lead to this need, such as ALS or spinal cord injury, and how many patients survive one year…