Hereditary motor neuron disease
MONDO:0024257An instance of motor neuron disease that is caused by an inherited modification of the individual's genome.
Also known as: genetic anterior horn cell disease, genetic motor neuron disease, hereditary motor neuron disease
164 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
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Gene therapy zolgensma tested in kids with SMA who can sit but not stand
Disease control TerminatedThis phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety …
Phase: PHASE1 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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ALS drug trial halted: safety data sought from 54 patients
Disease control TerminatedThis study tested a new drug called VRG50635 in 54 people with ALS (Lou Gehrig's disease). The main goal was to check if the drug is safe and how the body processes it. The trial was stopped early, but researchers were looking for side effects and changes in disease progression.
Phase: PHASE1 • Sponsor: Verge Genomics • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Gut hormone shot aims to strengthen fragile bones in kids with muscle diseases
Knowledge-focused TerminatedThis study tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…
Phase: NA • Sponsor: University of Copenhagen • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:08 UTC