Neuromuscular Disease Therapeutics Market to Reach US$ 45.62 Billion by 2033 as Gene Therapies and RNA-based Treatments Redefine Patient Outcomes

AUSTIN, Texas and TOKYO, Oct. 7, 2025 /PRNewswire/ -- The Neuromuscular Disease Therapeutics Market is entering a period of accelerated growth, supported by breakthroughs in gene therapy, antisense oligonucleotides (ASOs), monoclonal antibodies, and small-molecule modulators. According to DataM Intelligence, the Neuromuscular Disease Therapeutics Market Size reached US$ 11.89 billion in 2023, rose to US$ 13.70 billion in 2024, and is projected to reach US$ 45.62 billion by 2033, growing at a CAGR of 14.4% during 2025–2033.

Neuromuscular diseases-including amyotrophic lateral sclerosis (ALS), muscular dystrophies, spinal muscular atrophy (SMA), and myasthenia gravis-impact millions globally, with limited curative options historically. Advances in genomic medicine, targeted biologics, and digital diagnostics are reshaping the treatment landscape.

Global leaders such as Novartis, Biogen, AbbVie, AstraZeneca, Argenx, Takeda, and Sarepta Therapeutics are pioneering next-generation therapeutics. With strong regulatory backing, particularly from the U.S. FDA and Japan's PMDA, and rising investment in rare disease research, the neuromuscular therapeutics space is poised for sustained double-digit growth.

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Market Segmentation Analysis

By Therapy

Gene Therapies dominated with US$ 4.6 billion in 2024. Novartis' Zolgensma® for SMA and Sarepta's gene therapies for Duchenne muscular dystrophy highlight the transformative role of single-dose, potentially curative treatments. By 2033, gene therapies are expected to contribute nearly 40% of total revenues.

Antisense Oligonucleotides (ASOs), valued at US$ 3.2 billion in 2024, continue to expand, with Biogen's Spinraza® leading SMA care and newer ASOs under development for ALS and myopathies.

Small-Molecule Splicing Modulators, worth US$ 2.1 billion in 2024, are expanding in SMA and myositis, offering oral administration advantages.

Monoclonal Antibodies & Complement Inhibitors generated US$ 2.8 billion in 2024, driven by Argenx's Vyvgart® for myasthenia gravis and AstraZeneca's rare disease-focused biologics portfolio.

By Disease Type

• Amyotrophic Lateral Sclerosis (ALS), valued at US$ 3.4 billion in 2024, remains a critical focus, with ongoing R&D in gene therapy and neuroprotective approaches.
• Spinal Muscular Atrophy (SMA), contributing US$ 3.2 billion in 2024, continues to expand with gene therapies, ASOs, and oral splicing modulators.
• Muscular Dystrophies, worth US$ 2.8 billion in 2024, benefit from Sarepta and Pfizer's pipelines in Duchenne therapies.
• Myasthenia Gravis, valued at US$ 2.1 billion, is seeing rapid adoption of monoclonal antibody treatments.

By Route of Administration

Intravenous infusion therapies dominated (US$ 6.2 billion in 2024), covering ASOs, monoclonal antibodies, and enzyme-based treatments.
Oral therapies accounted for US$ 4.1 billion, led by small-molecule splicing modulators.
Subcutaneous administration contributed US$ 2.7 billion, offering greater patient convenience.

Regional Insights:

United States

The U.S. neuromuscular disease therapeutics market, valued at US$ 6.5 billion in 2024, leads globally due to:

• Strong regulatory support through FDA orphan drug designations and accelerated approvals.
• High uptake of Zolgensma®, Spinraza®, and Vyvgart®.
• Expanding reimbursement frameworks for rare disease drugs.
• Recent update (2025): Biogen received FDA approval for a next-generation ASO targeting ALS progression.

Japan

Japan's market, valued at US$ 1.2 billion in 2024, is growing due to:

• Robust government initiatives to improve rare disease access under the Rare and Intractable Diseases Act.
• Increasing adoption of SMA and myasthenia gravis biologics.
• Recent highlight (2025): Nippon Shinyaku launched a new oral therapy for Duchenne muscular dystrophy, further strengthening Japan's domestic pipeline.

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Industry Trends & 2025 Developments

• Gene Therapy Momentum: Expansion beyond SMA and DMD into ALS and Charcot-Marie-Tooth.
• ASO Expansion: Novel ASOs with better safety and longer dosing intervals are entering trials.
• AI-driven Diagnostics: Machine learning models are improving early detection of neuromuscular disorders.
• M&A & Collaborations: In 2025, Takeda partnered with a U.S. biotech to co-develop gene therapies for rare myopathies.
• Patient-Centric Models: Digital health platforms are improving trial recruitment and therapy adherence.

Competitive Landscape

Novartis Pharmaceuticals Corporation

Novartis leads the SMA market with Zolgensma®, generating over US$ 2 billion annually. Its strong pipeline in gene therapy reinforces long-term leadership.

Biogen Inc.

Biogen dominates the ASO space with Spinraza® and is advancing ALS-specific RNA-based therapies. Its 2025 approval strengthens its neurology franchise.

Sarepta Therapeutics

Sarepta is pioneering Duchenne muscular dystrophy gene therapies. In 2024, its revenues crossed US$ 1.5 billion, with new approvals expected in 2026.

Argenx

Argenx is revolutionizing myasthenia gravis care with Vyvgart®, which achieved blockbuster status in 2024. Its complement inhibitor pipeline is expanding into other neuromuscular diseases.

Takeda & Nippon Shinyaku

Japanese leaders are focusing on Duchenne and rare myopathies, leveraging domestic and global collaborations.

Strategic Outlook

The Neuromuscular Disease Therapeutics Market is undergoing a paradigm shift, defined by:

1. Gene Therapy Expansion – Single-dose curative treatments will reshape market dynamics.
2. RNA-based Leadership – ASOs and splicing modulators are expanding into ALS, SMA, and myopathies.
3. Monoclonal Antibodies Growth – Targeted therapies for myasthenia gravis and related disorders gaining rapid uptake.
4. Regional Access – The U.S. and Japan driving innovation adoption through strong regulatory and reimbursement frameworks.

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Conclusion

The Neuromuscular Disease Therapeutics Market, projected to rise from US$ 13.70 billion in 2024 to US$ 45.62 billion by 2033, is one of the fastest-growing segments in rare disease care. With Novartis, Biogen, Sarepta, and Argenx leading breakthroughs, the decade ahead will be defined by gene therapy milestones, RNA innovations, and global collaborations.

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SOURCE DataM Intelligence 4 Market Research LLP