NEUROMUSCULAR DISEASE

This study tests whether starting non-invasive ventilation (a breathing machine) earlier than usual helps people with ALS. Researchers will use a prediction tool to find those at high risk of breathing problems within 6 months. 48 participants will be randomly assigned to early N…

This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…

This early study tests a brain-computer implant that reads brain signals to help people with severe paralysis (from ALS, stroke, or spinal cord injury) communicate. The device aims to decode imagined speech and allow control of computers. Only 2 participants will be enrolled to c…

This study is testing a new, painless device called electrical impedance myography (EIM) to measure muscle health. Researchers will use it on healthy volunteers and people with neuromuscular diseases to see how well it works compared to standard tests like ultrasound and nerve st…

This study uses a safe, radiation-free device called electrical impedance tomography (EIT) to take pictures of the lungs in infants, children, and young adults up to age 25. Researchers will compare images from patients with chronic lung or heart conditions to healthy controls to…

This study looks at whether using a swallow gel or a semi-solid vehicle can help people with neurological conditions swallow pills more easily and safely. Researchers will observe 500 participants, including patients with swallowing disorders and healthy volunteers, as they swall…