Spinal muscular atrophy, type II

Researchers test GCB-001, a gene therapy given as a single injection into the spinal fluid, in children aged 2 to 12 with type 2 spinal muscular atrophy who can sit alone but cannot walk. The trial checks safety and whether the treatment improves motor skills over 12 months. It i…

This trial tests the long-term safety and effectiveness of apitegromab, an investigational antibody that blocks myostatin, a protein that limits muscle growth. People with type 2 or type 3 spinal muscular atrophy who completed earlier apitegromab studies can join. Researchers wil…

This early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …

This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINR…

Researchers are testing a muscle biopsy method that uses mild electrical stimulation to sample the junction where nerves meet muscle. The goal is to see if this technique helps distinguish between inclusion body myositis and motor neuron disease, which can look alike. The study i…

This study aims to find early markers of Parkinson's disease, multiple system atrophy, and Lewy body dementia by analyzing samples from the nose, blood, and urine. Researchers will compare results from 180 people with these conditions and healthy volunteers. The goal is to improv…

This study follows 1000 people with upper motor neuron disease who are about to receive the Cionic Neural Sleeve. The sleeve uses electrical stimulation to help muscles contract at the right time during movement. Researchers will track changes in health-related quality of life us…