How has the nucleic acid and gene therapies in neuromuscular disorders market evolved, and where is it heading next?
The nucleic acid and gene therapies in neuromuscular disorders market size has grown strongly in recent years. It will grow from $6.84 billion in 2024 to $7.52 billion in 2025 at a compound annual growth rate (CAGR) of 9.9%. The growth in the historic period can be attributed to increase in regulatory approvals, demand for nucleic acid therapeutics, demand for more precise and effective therapies, demand for effective gene therapies, and increase in the number of gene therapy-based discoveries.
The nucleic acid and gene therapies in neuromuscular disorders market size is expected to see strong growth in the next few years. It will grow to $10.87 billion in 2029 at a compound annual growth rate (CAGR) of 9.7%. The growth in the forecast period can be attributed to increasing prevalence of genetic disorders, increasing investments in research and development, growing demand for precision medicine, rising approval of gene therapy products, and increasing prevalence of chronic disorders. Major trends in the forecast period include advances in patient care, high performance chromatography, innovative solutions for conditions, advancements in gene editing technologies, and availability of nucleic acid extraction technology.
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What are the key drivers behind the rapid expansion of the nucleic acid and gene therapies in neuromuscular disorders market?
The increasing prevalence of genetic disorders is expected to propel the growth of the nucleic acid and gene therapies in neuromuscular disorders market going forward. Genetic disorders are diseases caused by abnormalities in an individual’s DNA, either inherited or resulting from mutations. The increase in genetic disorders is due to factors such as genetic mutations, environmental influences, lifestyle changes, and improved diagnostic capabilities. Nucleic acid and gene therapies help genetic disorders by correcting or replacing defective genes in neuromuscular conditions to restore normal function. For instance, in May 2024, according to Cure SMA, a US-based non-profit organization, In the United States, approximately 9,000 to 9,500 individuals are currently living with Spinal Muscular Atrophy (SMA). Among the total population, 37% have Type 2 SMA. The condition has an estimated incidence rate of 1 in 15,000 births. Therefore, the increasing prevalence of genetic disorders is driving the growth of the nucleic acid and gene therapies in neuromuscular disorders market.
What is the segmentation for the nucleic acid and gene therapies in neuromuscular disorders market?
The nucleic acid and gene therapies in neuromuscular disorders market covered in this report is segmented –
1) By Disorder: Motor Neuron Diseases, Neuropathies, Neuromuscular Junction Disorders, Myopathies Including Muscular Dystrophies
2) By Therapy: Adeno-Associated Viruses Gene Therapy, Postnatal Gene Therapy, Spinal Muscular Atrophy
3) By Application: Hospitals, Specialty Clinics, Ambulatory Surgery Centers
Subsegments:
1) By Motor Neuron Diseases: Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), Primary Lateral Sclerosis (PLS)
2) By Neuropathies: Charcot-Marie-Tooth Disease (CMT), Hereditary Sensory and Autonomic Neuropathy (HSAN), Peripheral Neuropathies
3) By Neuromuscular Junction Disorders: Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome (LEMS)
4) By Myopathies Including Muscular Dystrophies: Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), Limb-Girdle Muscular Dystrophy (LGMD), Facioscapulohumeral Muscular Dystrophy (FSHD)
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Who are the most influential companies in the nucleic acid and gene therapies in neuromuscular disorders market?
Major companies operating in the nucleic acid and gene therapies in neuromuscular disorders market are Pfizer Inc., F. Hoffmann-La Roche Ltd., Bayer AG, Sanofi S.A, Novartis AG, Eli Lilly and Company, Astellas Pharma Inc, Biogen Inc, Vertex Pharmaceuticals Inc, BioMarin Pharmaceuticals Inc, Nippon Shinyaku Co Ltd, Sarepta Therapeutics Inc, Ionis Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc, Amicus Therapeutics Inc, Arrowhead Pharmaceuticals Inc, Regenxbio Inc, UniQure N.V., Généthon S.A, Axovant Gene Therapies Inc.
What are the most influential trends expected to drive the nucleic acid and gene therapies in neuromuscular disorders market forward?
Major companies are operating in the nucleic acid and gene therapies in neuromuscular disorders market, focusing on developing innovative therapies such as gene therapy to address the root causes of genetic neuromuscular conditions through targeted gene correction or replacement. Gene therapy refers to the treatment of diseases by introducing, altering, or repairing genetic material within a patient’s cells to correct or replace defective genes. For instance, in June 2023, Sarepta Therapeutics Inc., a US-based biotechnology company, received approval from the U.S. Food and Drug Administration for Elevidys, the first gene therapy for Duchenne Muscular Dystrophy, specifically for ambulatory pediatric patients aged 4 to 5 years. The therapy is administered as a single intravenous infusion, simplifying the treatment process compared to other therapies that may require ongoing infusions. ELEVIDYS delivers a gene that codes for a shortened form of dystrophin known as micro-dystrophin, which is essential for muscle function. This innovative approach addresses the underlying genetic defect by enabling muscle cells to produce a functional version of the dystrophin protein, thereby potentially halting or reversing disease progression.
What are the major regional insights for the nucleic acid and gene therapies in neuromuscular disorders market, and which region holds the top position?
North America was the largest region in the nucleic acid and gene therapies in neuromuscular disorders market in 2024. The regions covered in the nucleic acid and gene therapies in neuromuscular disorders market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
What Does The Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market Report 2025 Offer?
The nucleic acid and gene therapies in neuromuscular disorders market research report from The Business Research Company offers global market size, growth rate, regional shares, competitor analysis, detailed segments, trends, and opportunities.
Nucleic acid and gene therapies in neuromuscular disorders refer to innovative treatments that aim to correct or compensate for the genetic mutations underlying these diseases. Neuromuscular disorders (NMDs) are conditions that affect the muscles and nerves, often due to inherited genetic mutations. These therapies involve the use of nucleic acids or genetic material to repair, replace, or modulate gene expression, thereby addressing the root cause of the disorder.
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