Familial amyotrophic lateral sclerosis
MONDO:0005144An instance of amyotrophic lateral sclerosis that is caused by an inherited modification of the individual's genome.
Also known as: hereditary amyotrophic lateral sclerosis
39 clinical trials for this condition and its sub-types, 2 tagged with Familial amyotrophic lateral sclerosis itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Familial amyotrophic lateral sclerosis
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Spinocerebellar ataxia type 2 10 trials
1 sub-type
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Amyotrophic lateral sclerosis type 1 8 trials
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Amyotrophic lateral sclerosis 28 0 trials
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Amyotrophic lateral sclerosis type 3 0 trials
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Amyotrophic lateral sclerosis type 8 0 trials
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Amyotrophic lateral sclerosis type 9 0 trials
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4 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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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