Back to discover
Disease

Neuromuscular Diseases

Late-stage therapeutic developmentEmerging research
MeSHNeuromuscular Diseases
1
Publications
16
Clinical trials
2024
Latest publication
Latest activity
beta

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: Low
Key developments
  • 3 clinical trials expected to report results, the earliest in Q3 2027.
Upcoming Milestones
Clinical Milestones5View
Results posted 1
Results expected 3
Recently completed 1
Activity timeline5

Clinical trials

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

The current development programme across all trial phases.

Clinical programme
16
All trials
6
Active
9
Late-stage
6
Completed
Late-stage studies

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping

Phase 3 · Recruiting · Dyne Therapeutics

An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

Phase 3 · Active, not recruiting · Scholar Rock, Inc.

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Phase 3 · Recruiting · Intellia Therapeutics

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

Phase 3 · Active, not recruiting · Capricor Inc.

A Phase 3, Double-Blind, Randomized, Multicenter, Placebo-Controlled Study to Evaluate the Efficacy and Safety of TNX-102 SL Tablets Taken Daily at Bedtime in Patients With Fibromyalgia

Phase 3 · Completed · Tonix Pharmaceuticals, Inc.

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Phase 3 · Completed · Scholar Rock, Inc.
Recruiting

Personalized Exercise Training for People With Rare Neuromuscular Disorders: a Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study to Assess the Effects on Physical Function From Personalized Strength and Balance Exercise in a Rehabilitation Setting.

N/A · Recruiting · Oslo University Hospital

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping

Phase 3 · Recruiting · Dyne Therapeutics

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Phase 3 · Recruiting · Intellia Therapeutics
Recently completed

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Phase 3 · Completed · Scholar Rock, Inc.

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Phase 3 · Completed · PTC Therapeutics

Assessing Air Travel Safety in Neuromuscular Dystrophy: Standard Versus Prolonged High Altitude Simulation Tests

N/A · Completed · The Hospital for Sick Children

A Phase 3, Double-Blind, Randomized, Multicenter, Placebo-Controlled Study to Evaluate the Efficacy and Safety of TNX-102 SL Tablets Taken Daily at Bedtime in Patients With Fibromyalgia

Phase 3 · Completed · Tonix Pharmaceuticals, Inc.

Effectiveness of a Protocol of Respiratory Physiotherapy for Improvement or Maintenance of Respiratory Function, and Incidence of Respiratory Infections in Children With Neuromuscular Diseases, Compared With no Approach by Respiratory Physiotherapy of Respiratory Difficulties: A Randomized Clinical Trial With Two Groups in Parallel With Blinded Evaluator.

N/A · Completed · University of Seville

A Pilot Study To Evaluate the Effect of Retrovir (Zidovudine: AZT) in the Treatment of Human Immunodeficiency Virus (HIV) Associated Dementia and Neuromuscular Diseases

N/A · Completed · Glaxo Wellcome

Research activity

1 papers

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

Most influential

Therapeutic targeting of RNA for neurological and neuromuscular disease.

Genes & development · 2024 · 5 cites
Recent publications

Therapeutic targeting of RNA for neurological and neuromuscular disease.

Genes & development · 2024 · 5 cites
Major themes3
  • Nervous System Diseases1
  • Neuromuscular Diseases1
  • RNA1
Leading journals1
  • Genes & development1
Leading researchers3
  • Bubenik JL1
  • Scotti MM1
  • Swanson MS1
Affiliations (unnormalised)1
  • Center for NeuroGenetics1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A general term encompassing lower MOTOR NEURON DISEASE; PERIPHERAL NERVOUS SYSTEM DISEASES; and certain MUSCULAR DISEASES. Manifestations include MUSCLE WEAKNESS; FASCICULATION; muscle ATROPHY; SPASM; MYOKYMIA; MUSCLE HYPERTONIA, myalgias, and MUSCLE HYPOTONIA.

Identifiers
MeSHNeuromuscular Diseases
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.