Press release
Limb-Girdle Muscular Dystrophy Market Set for Strong Growth to USD 2.4 Billion by 2031, Led by North America's 41.3% Market Share | DataM Intelligence
The global limb-girdle muscular dystrophy market reached USD 1.3 billion in 2023 and is expected to reach USD 2.4 billion by 2031, growing at a CAGR of 7.4% during the forecast period 2024-2031.Market growth stems from rising prevalence of rare genetic disorders, advancements in gene therapies like CRISPR-based treatments, and expanding clinical pipelines for subtype-specific drugs such as BBP-418 and SRP-9003. Increased R&D investments, improved genetic diagnostics, and regulatory approvals for novel enzyme replacement and RNA therapies further propel expansion.
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United States: Key Industry Developments
✅ December 2025: Edgewise Therapeutics advanced their LGMD gene therapy candidate into Phase II trials, focusing on CAPN3 mutations for improved muscle preservation and patient mobility.
✅ October 2025: The FDA granted accelerated approval to a novel exon-skipping therapy from Sarepta Therapeutics for LGMD2D subtype, enabling earlier intervention and better functional outcomes.
✅ August 2025: NIH-funded consortium launched a national patient registry expansion to accelerate LGMD clinical trials and biomarker validation.
Asia Pacific / Japan: Key Industry Developments
✅ November 2025: Japan's National Center of Neurology and Psychiatry initiated a Phase I trial for AAV-based gene therapy targeting LGMD2I, supported by government rare disease funding.
✅ September 2025: Chinese biotech firm Innovent Biologics partnered with local genomics centers to launch expanded genetic testing programs for LGMD subtypes in tier-1 cities.
✅ July 2025: South Korea's Samsung Bioepis announced early-stage development of stem cell therapies for LGMD, emphasizing regenerative approaches amid rising regional investments.
Key Merges and Acquistions:
✅ Companies in the Limb-Girdle Muscular Dystrophy market pursued strategic partnerships and licensing deals to expand gene therapy pipelines for rare neuromuscular disorders.
✅ Biotech firms closed global collaboration agreements adding clinical- and pre-clinical-stage programs targeting muscle diseases, enhancing treatment portfolios.
✅ Investments in spin-out therapeutics from research hospitals included upfront payments and acquisition options, focusing on innovative LGMD-specific gene therapies.
Market Segmentation Analysis:
-By Type: Type 2 Leads Due to Higher Prevalence
Limb-Girdle Muscular Dystrophy Type 2 holds the largest share at 56%, more common as a recessive form affecting proximal muscles with progressive weakness.
Type 1 accounts for 44%, rarer autosomal dominant variant with slower progression and variable onset.
-By Treatment Type: Physical Therapy Dominates Symptom Management
Physical Therapy commands 34% market share, essential for maintaining muscle strength, flexibility, and delaying mobility loss in daily function.
Medication takes 25%, using corticosteroids to reduce inflammation; Occupational Therapy 15% aids fine motor skills; Respiratory and Cardiac Care 10% supports vital functions; Mobility Aids 8% enhance independence; Others 8% cover emerging therapies.
-By End-Users: Hospitals & Clinics Lead Comprehensive Care
Hospitals & Clinics hold 55% share, providing diagnostics, multidisciplinary teams, and specialized neuromuscular services.
Homecare follows at 30%, enabling ongoing therapy and monitoring for chronic management; Others 15% include specialty centers.
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Why is the Limb-Girdle Muscular Dystrophy Market Growing?
The rapid expansion of the Limb-Girdle Muscular Dystrophy (LGMD) market in 2026 is fueled by several critical factors:
-Advancements in Gene Therapy
Breakthroughs in CRISPR/Cas9 and other gene-editing technologies enable targeted corrections of genetic mutations causing LGMD subtypes, improving treatment efficacy.
Pipeline candidates like SRP-9003 and LION-101 are advancing toward approval, addressing unmet needs in this rare disease.
-Improved Diagnostics
Genetic testing and molecular diagnostics facilitate earlier detection, expanding the addressable patient population and supporting personalized therapies.
Rising awareness of neuromuscular disorders drives demand for tools like muscle biopsy and MRI imaging.
-Robust R&D Pipeline
Increasing investment in enzyme replacement, RNA-based therapies, and stem cell approaches accelerates innovation amid growing prevalence of genetic mutations.
Patient advocacy and clinical trials further bolster drug development pipelines.
-Regional Healthcare Expansion
Heightened funding in North America and Europe, alongside Asia-Pacific infrastructure growth, supports commercialization and access to novel treatments.
Regional Insights:
-North America leads the Limb-Girdle Muscular Dystrophy (LGMD) market with the highest share of 41.3% in 2024, driven by advanced healthcare infrastructure, high prevalence of genetic disorders, and strong adoption of gene therapies alongside significant clinical trial activity in the U.S., which holds 82.4% of the regional revenue.
-Europe follows as the second-largest region, benefiting from robust research ecosystems, favorable orphan drug regulations, and key markets like Germany, France, Italy, Spain, and the UK, though exact share figures trail North America's dominance amid growing pipeline developments.
-Asia Pacific ranks third, with China capturing 39.6% of the regional market in 2024 due to rapid healthcare improvements, expanding genetic testing, and local biotech innovations, while overall regional growth lags behind North America and Europe owing to varying infrastructure levels.
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Key Players:
Horizon Therapeutics plc | Teva Pharmaceuticals USA, Inc. | Pfizer Inc. | Viatris Inc. | Atamyo Therapeutics | Sarepta Therapeutics, Inc. | Asklepios BioPharmaceutical, Inc. | ML Bio Solutions, Inc. | Genethon | Edgewise Therapeutics
Highlights (Top 5 Key Players) for Limb-Girdle Muscular Dystrophy Market:
-Sarepta Therapeutics, Inc. develops gene therapy programs targeting specific LGMD subtypes like LGMD2E using AAV-based vectors to restore missing muscle proteins and improve mobility.
-Asklepios BioPharmaceutical, Inc. advances AB-1003 (LION-101), an AAV gene therapy for LGMD2I/R9, delivering the functional FKRP gene to stabilize muscle function in ongoing phase 1/2 trials.
-Atamyo Therapeutics focuses on gene therapies for LGMD, including candidates that address underlying genetic mutations to halt muscle degeneration and enhance patient quality of life.
-Genethon pioneers AAV vector-based gene therapies for rare dystrophies like LGMD, conducting preclinical and clinical research to correct defective genes in muscle cells.
-Edgewise Therapeutics targets muscle diseases including LGMD subtypes with small molecule therapeutics designed to improve muscle strength and prevent fibrosis progression.
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