Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market to Reach USD 19.42 Billion by 2034

Pune, India - December 2025 - The global Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market, valued at USD 6.84 billion in 2024, is projected to reach USD 19.42 billion by 2034, growing at a robust 11.0% CAGR (2025-2034), according to Exactitude Consultancy. Rapid advancements in AAV-based gene therapies, antisense oligonucleotides (ASOs), RNA-targeting technologies, and improved diagnostic pathways are driving strong market momentum.

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Market Summary
The market for Nucleic Acid and Gene Therapies in Neuromuscular Disorders is expanding rapidly as genetic insights reshape treatment strategies for conditions like Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease, and myotonic dystrophy. Therapeutic modalities-including AAV gene replacement, exon-skipping antisense oligonucleotides, RNA interference (RNAi), CRISPR gene editing, and next-generation viral and non-viral delivery systems-are demonstrating unprecedented clinical potential.

Increased newborn screening for SMA, rapid regulatory approvals, and rising investment from biotech companies are significantly accelerating market adoption. North America and Europe dominate due to strong research ecosystems, while Asia-Pacific emerges as a major growth region with expanding clinical trial infrastructure.

Key Takeaways
• 2024 Market Size: USD 6.84 Billion
• 2034 Forecast: USD 19.42 Billion
• CAGR: 11.0% (2025-2034)
• Strong demand for AAV and ASO-based therapies
• Increasing regulatory approvals driving commercial growth
• Asia-Pacific becoming a critical region for clinical trials & patient access

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Market Drivers
• Major breakthroughs in gene replacement & gene-editing therapies
• Rising prevalence of neuromuscular genetic disorders
• Expansion of newborn screening programs for early detection
• Growing investments from pharma/biotech companies in rare diseases
• Regulatory incentives for orphan drugs accelerating development

Segmentation Snapshot
By Therapy Type
• AAV-Based Gene Replacement Therapy
• Antisense Oligonucleotides (Exon Skipping, Splice Modulation)
• RNA Interference (RNAi) Therapies
• CRISPR Gene Editing & Genome Engineering
• mRNA-Based Therapies
• Non-Viral Vector Therapies

By Target Disorder
• Duchenne Muscular Dystrophy (DMD)
• Spinal Muscular Atrophy (SMA)
• Amyotrophic Lateral Sclerosis (ALS)
• Charcot-Marie-Tooth Disease
• Myotonic Dystrophy
• Others

By End User
• Hospitals
• Gene Therapy Centers
• Specialty Neuromuscular Clinics
• Research Institutes

By Region
• North America (Largest Market)
• Europe
• Asia-Pacific (Fastest Growing)
• Latin America
• Middle East & Africa

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Recent Developments
• FDA and EMA approvals for next-gen gene therapies in SMA and DMD.
• CRISPR-based therapeutic candidates entering early-stage trials.
• Innovations in non-viral delivery enabling safer genetic medicine.
• Expanded newborn screening improving early intervention timelines.

Expert Quote - Irfan Tamboli, Business Development Executive
"Genetic medicine is transforming care for neuromuscular disorders, offering therapies that target the root cause of disease. As gene and nucleic acid therapies advance, millions of patients are gaining access to life-changing treatments."

Conclusion
The Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market is set for strong expansion through 2034, driven by scientific breakthroughs, regulatory support, and rising global demand for disease-modifying treatments. Companies pioneering AAV gene therapies, CRISPR editing, ASOs, and advanced RNA platforms will define the next decade of neuromuscular care.

This report is also available in the following languages : Japanese (神経筋疾患市場における核酸・遺伝子治療), Korean (신경근 질환 시장에서의 핵산 및 유전자 치료), Chinese (神经肌肉疾病核酸和基因疗法市场), French (Marché des thérapies par acides nucléiques et gènes dans les troubles neuromusculaires), German (Markt für Nukleinsäure- und Gentherapien bei neuromuskulären Erkrankungen), and Italian (Mercato delle terapie geniche e degli acidi nucleici nei disturbi neuromuscolari), etc.

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