Congenital Muscular Dystrophy Market

Market Overview:

The 7 major congenital muscular dystrophy markets reached a value of USD 1,553.2 Million in ​2024​. Looking forward, IMARC Group expects the 7MM to reach USD 2,123.0 Million by ​2035​, exhibiting a growth rate (CAGR) of 2.88% during ​2025-2035​.

The congenital muscular dystrophy market has been comprehensively analyzed in IMARC's new report titled "Congenital Muscular Dystrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast ​2025-2035​". Congenital muscular dystrophy is a collection of genetic conditions where muscles grow weaker and degenerate progressively from birth. It occurs in both genders and, if left untreated, can cause serious motor disability, contractures of the joints, respiratory issues, and sometimes even mental retardation. Congenital muscular dystrophy is variable in severity based on the particular gene mutation and some types may increase their severity more quickly than others. Early presentation may be with hypotonia (low muscle tone), late motor milestones, and feeding or respiratory difficulties. Diagnoses of CMD are made based on genetic testing, muscle biopsy, and imaging. Genetic testing is an essential part in determining mutations within genes like LAMA2, COL6A1, and FKRP, which are most frequently linked to CMD subtypes. Muscle biopsies can demonstrate characteristic histological alterations, including fibrosis and decreased dystrophin expression, whereas imaging tests such as MRI can assist in determining the degree of muscle involvement. Furthermore, serum creatine kinase (CK) levels are usually increased, acting as a preliminary marker of muscle disease.

The increasing prevalence of genetic mutations associated with congenital muscular dystrophy (CMD), disrupting the structure and function of muscles, is the prime driver of the CMD market. As a result of widening access to genetic testing, the rate of early diagnosis is rising, enabling better disease management and treatment. Greater research funding and clinical trials have also followed increasing awareness among patient organizations and healthcare professionals. The advent of advanced gene therapies, including CRISPR gene editing and viral vector gene replacement, is revolutionizing the CMD treatment landscape with potential long-term gains. Besides this, antisense oligonucleotide administration, capable of modulating gene expression to allow partial restoration of protein function, is expanding avenues for treatment. Beyond this, new pharmacologic approaches, like muscle-building compounds and regenerative medicine, are being explored to reverse muscle degeneration. The growing use of multidisciplinary care models, including physical therapy, respiratory care, and orthopedic care, is also contributing to enhanced patient outcomes, further driving market growth.

IMARC Group's new report provides an exhaustive analysis of the congenital 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, the United States has the largest patient pool for congenital 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 congenital muscular dystrophy market in any manner.

Recent Developments:

• In October 2024, Modalis Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MDL-101, an experimental therapy targeting congenital muscular dystrophy type 1A (LAMA2-CMD). This designation aims to support the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S.

Key Highlights:

• CMD affects approximately 1 to 9 individuals per 100,000 worldwide.
• While CMD occurs globally, specific prevalence rates can vary by region. For instance, in Italy, the prevalence is estimated at 1 in 16,000 individuals.
• CMD affects males and females equally, as the inheritance patterns are typically autosomal recessive or dominant, not linked to sex chromosomes.
• The prognosis of CMD varies depending on the subtype and severity. Life-threatening complications often arise from respiratory insufficiency and brain pathology.
• CMD encompasses a heterogeneous group of disorders caused by mutations in at least 15 different genes, affecting various components of muscle cells, including the extracellular matrix, dystrophin-associated glycoprotein complex, endoplasmic reticulum, and nuclear envelope.

Drugs:

Tideglusib, an investigational GSK-3β inhibitor, has shown promise in treating congenital myotonic dystrophy (CDM1). In the REACH-CDM Phase 2/3 trial, it demonstrated significant improvements in cognitive performance and motor function, with a favorable safety profile. AMO Pharma is advancing its development for CDM1 treatment.

MDL-101 is an experimental therapy for congenital muscular dystrophy type 1A (LAMA2-CMD). Utilizing CRISPR-based epigenome editing, it upregulates the LAMA1 gene to compensate for LAMA2 deficiency, aiming to provide a durable, one-time treatment. The FDA has granted it Orphan Drug and Rare Pediatric Disease designations.

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 congenital muscular dystrophy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the congenital 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 congenital 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 congenital 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 congenital 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 congenital 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 congenital muscular dystrophy across the seven major markets?
• What is the number of prevalent cases (​​2019-2035​​) of congenital muscular dystrophy by age across the seven major markets?
• What is the number of prevalent cases (​​2019-2035​​) of congenital muscular dystrophy by gender across the seven major markets?
• How many patients are diagnosed (​​2019-2035​​) with congenital muscular dystrophy across the seven major markets?
• What is the size of the congenital 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 congenital muscular dystrophy?
• What will be the growth rate of patients across the seven major markets?

Congenital 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 congenital 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 congenital muscular dystrophy market?
• What are the key regulatory events related to the congenital muscular dystrophy market?
• What is the structure of clinical trial landscape by status related to the congenital muscular dystrophy market?
• What is the structure of clinical trial landscape by phase related to the congenital muscular dystrophy market?
• What is the structure of clinical trial landscape by route of administration related to the congenital muscular dystrophy market?