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
176 clinical trials for this condition and its sub-types, 1 tagged with Hereditary motor neuron disease itself.
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Sub-types of Hereditary motor neuron disease
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Spinal muscular atrophy 107 trials · 117 incl. sub-types
19 sub-types
- Proximal spinal muscular atrophy 14 trials · 42 incl. sub-types Sub-types →
- Autosomal recessive distal spinal muscular atrophy 1 2 trials
- Bulbospinal muscular atrophy 1 trial Sub-types →
- X-linked distal spinal muscular atrophy type 3 0 trials
- Adult-onset proximal spinal muscular atrophy, autosomal dominant 0 trials
- Autosomal recessive distal spinal muscular atrophy 2 0 trials
- Infantile-onset X-linked spinal muscular atrophy 0 trials
- Neuronopathy, distal hereditary motor, autosomal dominant 0 trials Sub-types →
- Neuronopathy, distal hereditary motor, autosomal recessive 3 0 trials
- Neuronopathy, distal hereditary motor, autosomal recessive 4 0 trials
- Neuronopathy, distal hereditary motor, autosomal recessive 5 0 trials
- Scapuloperoneal spinal muscular atrophy, autosomal dominant 0 trials
- Scapuloperoneal spinal muscular atrophy, autosomal recessive 0 trials
- Spinal muscular atrophy type 0 0 trials
- Spinal muscular atrophy with respiratory distress type 2 0 trials
- Spinal muscular atrophy, Ryukyuan type 0 trials
- Spinal muscular atrophy, facioscapulohumeral type 0 trials
- Spinal muscular atrophy, segmental 0 trials
- Spinal muscular atrophy-progressive myoclonic epilepsy syndrome 0 trials
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Familial amyotrophic lateral sclerosis 2 trials · 29 incl. sub-types
30 sub-types
- Spinocerebellar ataxia type 2 10 trials Sub-types →
- Amyotrophic lateral sclerosis type 1 8 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 1 5 trials
- Amyotrophic lateral sclerosis type 10 3 trials
- Amyotrophic lateral sclerosis type 4 1 trial
- Amyotrophic lateral sclerosis type 6 1 trial
- Amyotrophic lateral sclerosis type 7 1 trial
- Amyotrophic lateral sclerosis 26 with or without frontotemporal dementia 0 trials
- Amyotrophic lateral sclerosis 27, juvenile 0 trials
- Amyotrophic lateral sclerosis 28 0 trials
- Amyotrophic lateral sclerosis type 11 0 trials
- Amyotrophic lateral sclerosis type 12 0 trials
- Amyotrophic lateral sclerosis type 15 0 trials
- Amyotrophic lateral sclerosis type 18 0 trials
- Amyotrophic lateral sclerosis type 19 0 trials
- Amyotrophic lateral sclerosis type 20 0 trials
- Amyotrophic lateral sclerosis type 21 0 trials
- Amyotrophic lateral sclerosis type 22 0 trials
- Amyotrophic lateral sclerosis type 23 0 trials
- Amyotrophic lateral sclerosis type 3 0 trials
- Amyotrophic lateral sclerosis type 8 0 trials
- Amyotrophic lateral sclerosis type 9 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 2 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 3 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 4 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 5 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 6 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 7 0 trials
- Frontotemporal dementia and/or amyotrophic lateral sclerosis 8 0 trials
- Juvenile amyotrophic lateral sclerosis 0 trials Sub-types →
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Lateral sclerosis 24 trials
2 sub-types
- Primary lateral sclerosis, adult, 1 1 trial
- Juvenile primary lateral sclerosis 0 trials
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Riboflavin transporter deficiency 1 trial · 6 incl. sub-types
3 sub-types
- Progressive bulbar palsy 5 trials Sub-types →
- Brown-Vialetto-van Laere syndrome 1 0 trials
- Brown-Vialetto-van Laere syndrome 2 0 trials
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Distal hereditary motor neuropathy 0 trials · 4 incl. sub-types
3 sub-types
- Neuronopathy, distal hereditary motor, autosomal recessive 0 trials · 4 incl. sub-types Sub-types →
- X-linked distal spinal muscular atrophy type 3 0 trials
- Neuronopathy, distal hereditary motor, autosomal dominant 0 trials Sub-types →
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ALS2-related motor neuron disease 0 trials
3 sub-types
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2 sub-types
Most studied deeper sub-types
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Spinal injection drug targets genetic cause of ALS and dementia
Disease control Stopped earlyResearchers are testing repeated doses of an experimental drug called WVE-004 in adults who have ALS, frontotemporal dementia, or both, linked to a mutation in the C9orf72 gene. Participants receive the drug by spinal injection every 12 weeks for up to 96 weeks. The study tracks …
Phase 1/2 • Sponsor: Wave Life Sciences USA, Inc. • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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Gene therapy zolgensma tested in kids with SMA who can sit but not stand
Disease control Stopped earlyThis 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 1 • 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 Stopped earlyThis 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 1 • 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 Stopped earlyThis 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…
Sponsor: University of Copenhagen • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:08 UTC