NEUROMUSCULAR DISEASES (NMD)

This study tests a single dose of a gene therapy called NTLA-2001 in 60 people with a rare inherited nerve disease (ATTRv-PN). The goal is to see if it can safely reduce harmful protein buildup and slow nerve damage compared to a placebo. Participants must have a confirmed diagno…

This study tests a special fabric shoulder brace designed to mimic muscle action. Researchers want to see if it helps people with neuromuscular disorders (like muscular dystrophy or ALS) move their arms and hands better. About 30 participants will be evaluated with and without th…

This study is testing whether whole-body electrical muscle stimulation (WB-EMS) can help adults with neuromuscular diseases like ALS, muscular dystrophy, and myasthenia gravis exercise safely. Because these conditions make normal exercise difficult, WB-EMS uses electrical pulses …

This study tests a new home-based program that uses a virtual reality treadmill to help children aged 6 to 17 with conditions like cerebral palsy, neuromuscular diseases, or obesity improve their walking. The program involves at least three 30-minute sessions per week combining w…

This study is creating a large collection of blood, skin, and spinal fluid samples, along with health information, from 2,500 adults with neuromuscular diseases like ALS, neuropathy, and muscular dystrophy. The goal is to provide these resources to researchers worldwide to speed …

This study looks back at the last 10 years to understand how many people in Denmark use a breathing machine at home through a tube in their throat. Researchers will examine which diseases lead to this need and how many patients survive one year after starting. The goal is to gath…

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…