Amyotrophic lateral sclerosis type 6

MONDO:0011951

Any amyotrophic lateral sclerosis in which the cause of the disease is a mutation in the FUS gene.

Also known as: ALS6, FUS amyotrophic lateral sclerosis, amyotrophic lateral sclerosis caused by mutation in FUS, amyotrophic lateral sclerosis 6 with or without frontotemporal dementia

22 clinical trials for this condition and its sub-types, 1 tagged with Amyotrophic lateral sclerosis type 6 itself.

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Where it sits in the disease tree

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Part of

↑ Familial amyotrophic lateral sclerosis (29) ↑ Frontotemporal dementia with motor neuron disease (19)

Including sub-types (22) Tagged with Amyotrophic lateral sclerosis type 6 (1)

Not yet finished but already full! 1

Promising Gene-Targeting drug for ALS enters final trial phase

Disease control Ongoing

This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection. This i…

Phase 3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control

Last updated Jul 23, 2026 00:00 UTC

Amyotrophic lateral sclerosis type 6

Where it sits in the disease tree

Promising Gene-Targeting drug for ALS enters final trial phase