Limb-Girdle Muscular Dystrophy Market

Market Overview:

The 7 major limb-girdle muscular dystrophy markets reached a value of USD 612.1 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 865.0 Million by ​2035​, exhibiting a growth rate (CAGR) of 3.20% during ​2025-2035​.

The limb-girdle muscular dystrophy market has been comprehensively analyzed in IMARC's new report titled "Limb-Girdle Muscular Dystrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast ​2025-2035​". LGMD is a heterogeneous group of inherited neuromuscular diseases with progressive muscle weakness and degeneration, mainly involving the muscles of the shoulders, hips, and limbs. The condition may present at any age, from childhood to adulthood, and its severity depends on the type of genetic mutation. While in some, there is slow progression of symptoms, in others, there is rapid muscle wasting with severe disability. Early symptoms usually involve climbing stairs, getting up from sitting, falling frequently, and fatigue of muscles. With advancing disease, individuals may have joint contractures, rigidity of muscles, and cardiac and respiratory complications. Diagnosis includes genetic testing, muscle biopsy, electromyography (EMG), and measurement of creatine kinase (CK) levels to establish muscle damage and the genetic subtype. The heterogeneity of LGMD requires an accurate diagnosis for the selection of appropriate management strategies and potential participation in emerging clinical trials and gene therapy interventions.

The Limb-Girdle Muscular Dystrophy (LGMD) market is being driven largely by the rising incidence of genetic mutations that result in progressive muscle degeneration. The increasing awareness of neuromuscular disorders, combined with advances in genetic testing, is enabling early diagnosis and enhancing patient outcomes. Furthermore, the increased number of research studies aimed at creating gene therapies and new treatment methods is driving market growth. The advent of gene-editing tools like CRISPR and adeno-associated virus (AAV)-facilitated gene therapies promises new ways of correcting faulty genes that cause LGMD. Increased government and private funding for research in rare diseases is also speeding up the pace of clinical trials to identify effective therapy. Accessibility to assistive devices, physiotherapy, and supportive interventions is improving patient life quality, enhancing the need for expert care. Pharmaceuticals are investing aggressively in disease-modifying therapies, such as enzyme replacement therapies and regenerative medicine, in order to meet the unmet medical needs of LGMD patients. The increased collaboration among biotech companies and research institutions will propel considerable growth in the LGMD market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the limb-girdle muscular dystrophy market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, Europe has the largest patient pool for limb-girdle muscular dystrophy and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the limb-girdle muscular dystrophy market in any manner.

Recent Developments:

• In March 2024, regulatory bodies in France and Italy approved Atamyo Therapeutics' Phase 1b clinical trial for ATA-200, a gene therapy for LGMD type 2C/R5. The trial focuses on evaluating the safety and efficacy of a single-dose treatment in children aged 6-11, with patient dosing anticipated to begin in the third quarter of 2024.
• In September 2023, Atamyo Therapeutics' Phase 1/2 clinical trial for ATA-100, designed for LGMD type 2I/R9, was permitted to advance to its second cohort following encouraging early results. Initial data indicated a reduction in creatine kinase levels and improvements in muscle function, demonstrating promising therapeutic potential.
• In November 2024, researchers developed a novel gene therapy technique called "StitchR," which facilitates the delivery of large genes in animal models. This method enables gene fragments to merge inside the cell, effectively restoring essential proteins. While still in experimental stages, this innovation could pave the way for advanced treatments for LGMD.

Key Highlights:

• LGMD arises from mutations in genes like CAPN3 and DYSF, impairing muscle proteins. Over 30 subtypes exist, each with distinct genetic origins.
• About 90% of LGMD cases follow an autosomal recessive pattern, requiring two mutated gene copies, while 10% are autosomal dominant, needing only one defective gene.
• LGMD affects approximately 1.63 per 100,000 people globally. Some subtypes, like LGMD2I, are more common in Northern Europe, while LGMD2B is prevalent in Middle Eastern populations.
• Onset ranges from childhood to adulthood, with some subtypes leading to severe disability within 20 years. Respiratory and cardiac complications contribute to reduced life expectancy.
• Gene therapies like Sarepta’s SRP-9003 show promise, while steroids, physical therapy, and assistive devices help manage symptoms. Clinical trials continue exploring novel therapeutic approaches.

Drugs:

Bidridistrogene xeboparvovec (SRP-9003) is an investigational gene therapy by Myonexus Therapeutics for limb-girdle muscular dystrophy type 2E. It employs an AAVrh74 vector to deliver a functional SGCB gene, aiming to restore beta-sarcoglycan expression and enhance muscle function. Currently in late-stage development, it has received orphan drug and fast track designations.

MYO-201 is an experimental gene therapy developed by Myonexus Therapeutics for limb-girdle muscular dystrophy. It is designed to deliver a functional dystrophin-associated gene using an AAV vector, with the goal of improving muscle stability and function.

Patidistrogene bexoparvovec (SRP-9005), developed by Myonexus Therapeutics and Sarepta Therapeutics, is a gene therapy candidate for limb-girdle muscular dystrophy type 2D. It utilizes an AAVrh74 vector to introduce a functional SGCA gene, aiming to restore alpha-sarcoglycan protein levels and enhance muscle performance.

Ribitol, from ML Bio Solutions, is an oral therapy under investigation for limb-girdle muscular dystrophy type 2I. It functions as a metabolic precursor to improve alpha-dystroglycan glycosylation, potentially strengthening muscle integrity and delaying disease progression.

AB-1003, developed by Asklepios BioPharmaceutical, is a gene therapy for limb-girdle muscular dystrophy. Using an AAV vector, it delivers a therapeutic gene to restore protein expression, supporting muscle function and slowing disease advancement.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: ​2025-2035​

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the limb-girdle muscular dystrophy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the limb-girdle muscular dystrophy market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current limb-girdle muscular dystrophy marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the limb-girdle muscular dystrophy market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the limb-girdle muscular dystrophy market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the limb-girdle muscular dystrophy market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​​2019-2035​​) of limb-girdle muscular dystrophy across the seven major markets?
• What is the number of prevalent cases (​​2019-2035​​) of limb-girdle muscular dystrophy by age across the seven major markets?
• What is the number of prevalent cases (​​2019-2035​​) of limb-girdle muscular dystrophy by gender across the seven major markets?
• How many patients are diagnosed (​​2019-2035​​) with limb-girdle muscular dystrophy across the seven major markets?
• What is the size of the limb-girdle muscular dystrophy patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (​2025-2035​) across the seven major markets?
• What are the key factors driving the epidemiological trend of limb-girdle muscular dystrophy?
• What will be the growth rate of patients across the seven major markets?

Limb-Girdle Muscular Dystrophy: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for limb-girdle muscular dystrophy drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the limb-girdle muscular dystrophy market?
• What are the key regulatory events related to the limb-girdle muscular dystrophy market?
• What is the structure of clinical trial landscape by status related to the limb-girdle muscular dystrophy market?
• What is the structure of clinical trial landscape by phase related to the limb-girdle muscular dystrophy market?
• What is the structure of clinical trial landscape by route of administration related to the limb-girdle muscular dystrophy market?