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Disease

Motor Neuron Disease

Late-stage therapeutic developmentEmerging research
MeSHMotor Neuron Disease
2
Publications
23
Clinical trials
2024
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 5 clinical trials expected to report results, the earliest in Q2 2027.
  • 1 industry development reported.
Upcoming Milestones
Clinical Milestones8View
Results posted 2
Results expected 5
Other updates 1
Industry & Market1View
Activity timeline9

Clinical trials

16 sponsors · 1 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
23
All trials
6
Active
18
Late-stage
6
Completed
Late-stage studies

Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial

Phase 2/3 · Recruiting · University of Edinburgh

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

Phase 3 · Recruiting · Prilenia

HEALEY ALS Platform Trial

Phase 2/3 · Recruiting · Merit E. Cudkowicz, MD

Randomized Double-blind Controlled Exploratory Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

Phase 4 · Recruiting · Peking University Third Hospital

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Phase 3 · Active, not recruiting · Biogen

A Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudilast) Followed by Open-Label Extension Phase in Subjects With Amyotrophic Lateral Sclerosis

Phase 2/3 · Active, not recruiting · MediciNova
Recently completed

Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations

Phase 4 · Completed · ChaodongWang

HEALEY ALS Platform Trial - Regimen G DNL343

Phase 2/3 · Completed · Merit E. Cudkowicz, MD

A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants With Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX)

Phase 3 · Completed · Amylyx Pharmaceuticals Inc.

A Double-blind, Randomized, Multicenter, Placebo-Controlled, Parallel, Phase III Clinical Trial to Evaluate the Efficacy and Safety of Lenzumestrocel(Neuronata-R® Inj.) in Patients With Amyotrophic Lateral Sclerosis

Phase 3 · Completed · Corestemchemon, Inc.

Multicenter, Open-label Extension Study Following the Studies MT-1186-A03 or A04 to Evaluate the Safety of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Phase 3 · Completed · Tanabe Pharma Corporation

Japanese Early-stage Clinical Trial of Ultra-high Dose Methylcobalamin for Amyotrophic Lateral Sclerosis: a Pivotal Phase 3 Randomized Controlled Study

Phase 3 · Completed · Eisai Co., Ltd.

Research activity

2 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20232024
Major themes8
  • Motor Neuron Disease2
  • Amyotrophic Lateral Sclerosis1
  • Brain Ischemia1
  • Dementia1
  • Endogenous Retroviruses1
  • HIV Infections1
  • Ischemic Stroke1
  • Parkinson Disease1
Leading journals2
  • Journal of neurology, neurosurgery, and psychiatry1
  • Molecular neurobiology1
Leading researchers7
  • Carr JM1
  • Dubowsky M1
  • Hägg S1
  • Mak JKL1
  • McMurran CE1
  • Rogers ML1
  • Theunissen F1
Affiliations (unnormalised)5
  • Centre for Molecular Medicine and Innovative Therapeutics1
  • College of Medicine and Public Health and Flinders Health and Medical Research Institute1
  • Department of Clinical Neurosciences1
  • Karolinska Institutet1
  • Perron Institute for Neurological and Translational Science1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Diseases characterized by a selective degeneration of the motor neurons of the spinal cord, brainstem, or motor cortex. Clinical subtypes are distinguished by the major site of degeneration. In AMYOTROPHIC LATERAL SCLEROSIS there is involvement of upper, lower, and brainstem motor neurons. In progressive muscular atrophy and related syndromes (see MUSCULAR ATROPHY, SPINAL) the motor neurons in the spinal cord are primarily affected. With progressive bulbar palsy (BULBAR PALSY, PROGRESSIVE), the initial degeneration occurs in the brainstem. In primary lateral sclerosis, the cortical neurons are affected in isolation. (Adams et al., Principles of Neurology, 6th ed, p1089)

Identifiers
MeSHMotor Neuron Disease
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.