DelveInsight’s “Myotonic Dystrophy Market Insights, Epidemiology and Market Forecast – 2034” report delivers an in-depth understanding of the Myotonic Dystrophy, historical and forecasted epidemiology as well as the Myotonic Dystrophy market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

 

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Key Takeaways from the Myotonic Dystrophy Market Report

• In January 2025, Dyne Therapeutics announced that the FDA granted Fast Track designation for DYNE-101, an investigational treatment for myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.
• Among the 7MM, EU4 and the UK accounted for nearly 353,191 diagnosed prevalent cases of myotonic dystrophy, and these cases are expected to increase during the forecast period (2024–2034).
• Among EU4 and the UK, Germany had the highest diagnosed prevalent population of myotonic dystrophy, with 8,938 cases, followed by the UK and France in 2023. On the other hand, Spain had the lowest diagnosed prevalent population in EU4 and the UK in 2023.
• In Japan, there were around 12,735 diagnosed prevalent cases of Myotonic Dystrophy in 2023. These cases are expected to increase at a significant CAGR.
• In 2023, in Japan, approximately 1,834 cases of individuals with myotonic dystrophy were associated with gastrointestinal symptoms, 1,605 cases with cardiac dysrhythmias, 1,414 cases with sleep disorders, and 3,566 cases with other comorbidities.
• In 2023, the highest proportion of myotonic dystrophy cases in the 7MM was estimated to be among adults, with 96,287 cases, compared to 9,075 cases among children.
• According to the analysis conducted by DelveInsight, among diagnosed cases of myotonic dystrophy, approximately 80% were of myotonic dystrophy type 1 and 20% of myotonic dystrophy type 2 within the US in 2023. This analysis indicates a higher prevalence of myotonic dystrophy type 1.
• The leading Myotonic Dystrophy Companies such as AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others.
• Promising Myotonic Dystrophy Therapies such as Tideglusib, AOC 1001 (del-desiran), Mexiletine, and others.

 

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Myotonic Dystrophy Epidemiology Segmentation in the 7MM

• Myotonic Dystrophy Diagnosed Prevalence cases
• Myotonic Dystrophy Type-specific cases
• Myotonic Dystrophy Age-specific cases
• Comorbidities associated with Myotonic Dystrophy

 

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Myotonic Dystrophy Emerging Drugs

• AMO-02 (tideglusib): AMO Pharma Limited

AMO-02 (tideglusib) is under development for congenital myotonic dystrophy (CDM) and potential use in other CNS, neuromuscular, and orphan indications. It acts by disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess GSK3β kinase levels. The US FDA granted FTD and RPDD status, while the UK granted innovative passport designation. Tideglusib, developed by AMO Pharma, exhibits a dual mechanism, showing promise in improving CNS and neuromuscular symptoms with minimal adverse effects. Expected approval in 2024 addresses the significant unmet needs of congenital DM.

 

• Pitolisant: Harmony Biosciences, LLC

Pitolisant, developed by Bioprojet Societe Civile de Recherche, acts as a selective antagonist/inverse agonist of histamine 3 (H3) receptors. Harmony Biosciences holds an exclusive license from Bioprojet for its development, manufacturing, and commercialization in the US. Harmony Biosciences is currently conducting a Phase II trial for type 1 diabetes patients. Pitolisant, a stimulant targeting histamine H3 receptors, is under investigation by Harmony Biosciences in a Phase II trial. While it has demonstrated efficacy in treating excessive daytime sleepiness in narcolepsy, its potential for addressing similar symptoms in DM1 patients requires further investigation.

 

Myotonic Dystrophy Treatment Market

Myotonic Dystrophy treatment encompasses various classifications within the disease spectrum. The primary focus of managing Myotonic Dystrophy involves the use of Mexiletine, Anti-hypersensitives, Hypolipidemic drugs, Anti-diabetic drugs, NSAIDs, Anti-constipation medications, PPIs, Ursodeoxycholic acid, and Vitamin D Supplements as required, with Mexiletine serving as the principal revenue source in the current treatment framework.

 

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Myotonic Dystrophy Market Outlook

Myotonic dystrophy is a dominantly inherited type of muscular dystrophy that affects the muscles and other body systems. The disease can lead patients to experience early cataracts, myotonia, muscle weakness/atrophy, fatigue, excessive daytime sleepiness, central/obstructive apnoea, respiratory failure, cardiac arrhythmia, insulin resistance, dysphagia, mood disorders, and others. Myotonic dystrophy is of two types; myotonic dystrophy type 1 is caused by the expansion of a CTG triplet repeat in DMPK, whereas the expansion of a CCTG tetramer causes myotonic dystrophy type 2 repeat in CNBP.

 

Scope of the Myotonic Dystrophy Market Report

• Coverage- 7MM
• Myotonic Dystrophy Companies- AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others.
• Myotonic Dystrophy Therapies- Tideglusib, AOC 1001 (del-desiran), Mexiletine, and others.
• Myotonic Dystrophy Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Myotonic Dystrophy Unmet Needs, KOL’s views, Analyst’s views, Myotonic Dystrophy Market Access and Reimbursement

 

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Table of Content

1 Key Insights

2 Report Introduction

3 Myotonic Dystrophy Market Overview at a Glance

4 Epidemiology and Market Methodology

5 Executive Summary

6 Key Events

7 Disease Background and Overview

8 Epidemiology and Patient Population

9 Patient Journey

10 Emerging Drugs

11 Myotonic Dystrophy: Seven Major Market Analysis

12 KOL Views

13 SWOT Analysis

14 Unmet Needs

15 Market Access

16 Appendix

17 DelveInsight Capabilities

18 Disclaimer

19 About DelveInsight

 

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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