Myotonic Dystrophy Market to Expand Significantly by 2034, States DelveInsight Report | Design Therapeutics, Enzerna, Juvena Therapeutics, Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences

The Key Myotonic Dystrophy Companies in the market include - Design Therapeutics, Enzerna, Juvena Therapeutics, Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences, AMO Pharma, Lupin, AMO Pharma, Dyne Therapeutics, Entrada Therapeutics, and others.

DelveInsight's "Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast-2034 report offers an in-depth understanding of the Myotonic Dystrophy, historical and forecasted epidemiology as well as the Myotonic Dystrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Some of the key facts of the Myotonic Dystrophy Market Report:

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The Myotonic Dystrophy market size was valued approximately USD 89 million in 2023 and is anticipated to grow with a significant CAGR of 18.4% during the study period (2020-2034)

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In April 2025, Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company pioneering a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs Trademark ), announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to delpacibart etedesiran (del-desiran) for treating myotonic dystrophy type 1 (DM1). This investigational therapy targets the underlying cause of DM1, a progressive, often fatal, and underrecognized neuromuscular condition with no approved treatments. Notably, del-desiran is the first DM1 investigational therapy to receive orphan status in Japan and has also been granted Breakthrough Therapy, Orphan Drug, and Fast Track designations by the U.S. FDA, as well as Orphan Designation by the European Medicines Agency (EMA).

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In March 2025, Treatment with DYNE-101 is showing promising improvements in muscle function - particularly finger function - in individuals with myotonic dystrophy type 1 (DM1), according to updated interim results from a Phase 1/2 clinical trial. Data from the ongoing ACHIEVE study (NCT05481879) revealed that DM1 patients receiving the investigational therapy were able to open and close their middle finger more quickly after six months, whereas those on placebo experienced slower performance. Additional improvements in motor skills and muscle strength were also observed in the treated group compared to the placebo group.

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In January 2025, Dyne Therapeutics announced that the FDA has granted Fast Track designation to DYNE-101, an investigational therapy for myotonic dystrophy type 1 (DM1). DYNE-101 is currently under evaluation in the ongoing Phase 1/2 ACHIEVE global clinical trial.

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In December 2024, PepGen Inc.- The purpose of this study is to investigate the effects of the investigational medicine PGN-EDODM1 and evaluate the safety and tolerability of multiple administrations of PGN-EDODM1 in individuals with myotonic dystrophy type 1 (DM1) compared to a placebo

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In December 2024, Avidity Biosciences Inc. is conducting a study that involves a screening period of up to 6 weeks, followed by a 54-week treatment phase, totaling approximately 60 weeks. Participants will be randomly assigned to receive intravenous infusions of either del-desiran or a placebo at the study site every 8 weeks, for a total of 7 doses. The last dose will be given at Week 48, with the final evaluation taking place at Week 54.

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In December 2024, Vertex Pharmaceuticals is conducting a Phase 1/2 randomized, double-blind, placebo-controlled trial to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 in adults with Myotonic Dystrophy Type 1, using both single- and multiple-dose escalation.

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In November 2024, ARTHEx Biotech S.L., a clinical-stage biotech company specializing in gene expression modulation, announced that the FDA has awarded Rare Pediatric Disease Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular condition. This designation comes after the company previously received Orphan Drug Designation (ODD) for ATX-01 in DM1 from both U.S. and European regulatory bodies.

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Within the 7MM, the EU4 and the UK accounted for approximately 353,191 diagnosed prevalent cases of Myotonic Dystrophy, with the number of cases projected to rise throughout the forecast period (2024-2034).

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Among the EU4 countries and the UK, Germany recorded the highest number of diagnosed prevalent Myotonic Dystrophy cases at 8,938 in 2023, followed by the UK and France. Conversely, Spain had the lowest diagnosed prevalent population within the EU4 and the UK that year.

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In 2023, Japan reported approximately 12,735 diagnosed prevalent cases of Myotonic Dystrophy, with these numbers projected to grow at a substantial CAGR.

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In 2023, Japan had roughly 1,834 myotonic dystrophy cases linked to gastrointestinal symptoms, 1,605 cases associated with cardiac dysrhythmias, 1,414 cases with sleep disorders, and 3,566 cases presenting other comorbidities.

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In 2023, adults accounted for the largest share of myotonic dystrophy cases in the 7MM, with an estimated 96,287 cases, compared to 9,075 cases among children.

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Key Myotonic Dystrophy Companies: Design Therapeutics, Enzerna, Juvena Therapeutics, Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences, AMO Pharma, Lupin, AMO Pharma, Dyne Therapeutics, Entrada Therapeutics, and others

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Key Myotonic Dystrophy Therapies: DM1 program, ENZ-001, JUV 161, DYNE-101, AOC 1001, Pitolisant, Tideglusib, Mexiletine, AMO-02, DYNE-101, ENTR-701, and others

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The Myotonic Dystrophy epidemiology based on gender analyzed that DM is more prevalent in adults (>18 years) than children (

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The Myotonic Dystrophy market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Myotonic Dystrophy pipeline products will significantly revolutionize the Myotonic Dystrophy market dynamics.

Myotonic Dystrophy Overview

Myotonic Dystrophy is a genetic disorder characterized by progressive muscle weakness and prolonged muscle contractions (myotonia). It affects multiple body systems, including the muscles, heart, eyes, and endocrine system. The condition is inherited and typically worsens over time, leading to difficulties with movement, coordination, and other complications such as cardiac issues and sleep disturbances. There are two main types-Type 1 (DM1) and Type 2 (DM2)-with DM1 being the more common and severe form.

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Myotonic Dystrophy Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Myotonic Dystrophy Epidemiology Segmentation:

The Myotonic Dystrophy market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalence of Myotonic Dystrophy

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Prevalent Cases of Myotonic Dystrophy by severity

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Gender-specific Prevalence of Myotonic Dystrophy

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Diagnosed Cases of Episodic and Chronic Myotonic Dystrophy

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Myotonic Dystrophy Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Myotonic Dystrophy market or expected to get launched during the study period. The analysis covers Myotonic Dystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Myotonic Dystrophy Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Myotonic Dystrophy Therapies and Key Companies

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DM1 program: Design Therapeutics

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ENZ-001: Enzerna

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JUV 161: Juvena Therapeutics

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DYNE-101: Dyne Therapeutics

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AOC 1001: Avidity Biosciences

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Pitolisant: Harmony Biosciences

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Tideglusib: AMO Pharma

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Mexiletine: Lupin

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AMO-02: AMO Pharma

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DYNE-101: Dyne Therapeutics

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ENTR-701: Entrada Therapeutics

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Scope of the Myotonic Dystrophy Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Myotonic Dystrophy Companies: Design Therapeutics, Enzerna, Juvena Therapeutics, Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences, AMO Pharma, Lupin, AMO Pharma, Dyne Therapeutics, Entrada Therapeutics, and others

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Key Myotonic Dystrophy Therapies: DM1 program, ENZ-001, JUV 161, DYNE-101, AOC 1001, Pitolisant, Tideglusib, Mexiletine, AMO-02, DYNE-101, ENTR-701, and others

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Myotonic Dystrophy Therapeutic Assessment: Myotonic Dystrophy current marketed and Myotonic Dystrophy emerging therapies

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Myotonic Dystrophy Market Dynamics: Myotonic Dystrophy market drivers and Myotonic Dystrophy market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Myotonic Dystrophy Unmet Needs, KOL's views, Analyst's views, Myotonic Dystrophy Market Access and Reimbursement

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Table of Contents

1. Myotonic Dystrophy Market Report Introduction

2. Executive Summary for Myotonic Dystrophy

3. SWOT analysis of Myotonic Dystrophy

4. Myotonic Dystrophy Patient Share (%) Overview at a Glance

5. Myotonic Dystrophy Market Overview at a Glance

6. Myotonic Dystrophy Disease Background and Overview

7. Myotonic Dystrophy Epidemiology and Patient Population

8. Country-Specific Patient Population of Myotonic Dystrophy

9. Myotonic Dystrophy Current Treatment and Medical Practices

10. Myotonic Dystrophy Unmet Needs

11. Myotonic Dystrophy Emerging Therapies

12. Myotonic Dystrophy Market Outlook

13. Country-Wise Myotonic Dystrophy Market Analysis (2020-2034)

14. Myotonic Dystrophy Market Access and Reimbursement of Therapies

15. Myotonic Dystrophy Market Drivers

16. Myotonic Dystrophy Market Barriers

17. Myotonic Dystrophy Appendix

18. Myotonic Dystrophy Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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