Myotonic Dystrophy Therapeutics Market Size is projected to experience robust growth, at a significant CAGR of 18.4% from 2020 to 2034, estimates DelveInsight

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 and the Myotonic Dystrophy market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

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Key Takeaways from the Myotonic Dystrophy Market Report

* In December 2024:- Avidity Biosciences Inc.- AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).
* In December 2024:- Lupin Ltd-This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to
* 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 Limited, Lupin Ltd., Harmony Biosciences, LLC, Avidity Biosciences Inc. and others.
* Promising Myotonic Dystrophy Therapies such as Mexiletine, AOC 1001 (del-desiran), Tideglusib, ATX-01, VX-670 , and others.

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Myotonic Dystrophy Epidemiology Segmentation in the 7MM

* Total Myotonic Dystrophy Diagnosed Prevalent Cases
* Myotonic Dystrophy Type-specific Cases
* Myotonic Dystrophy Age-specific Cases
* Comorbidities associated with Myotonic Dystrophy

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Myotonic Dystrophy Emerging Drugs

* Tideglusib (AMO-02): AMO Pharma

AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has the potential for use in additional CNS, neuromuscular, and other orphan indications. AM0-02 is a clinical-stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as myotonic dystrophy type 1 or Steinert disease. In cellular and animal models of myotonic dystrophy type 1, as well as in muscle biopsies from patients, the activity of glycogen synthase kinase 3 beta (GSK3Ss) has been shown to increase. AMO-02 is an inhibitor that has been shown to normalize levels of GSK3Ss in transgenic models and in ex vivo tissue samples in patients with myotonic dystrophy type 1 and to reduce levels of the mRNA that is pathogenic for myotonic dystrophy type 1.

* Delpacibart etedesiran (AOC-1001): Avidity Biosciences, Inc.

Delpacibart etedesiran (AOC-1001), also known as del-desiran, is a monoclonal Antibody Oligonucleotide Conjugate (AOC), is designed to address the root cause of myotonic dystrophy type 1 by reducing levels of a disease-related mRNA called DMPK in skeletal, cardiac, and smooth muscle. Del-desiran consists of a proprietary Monoclonal Antibody (mAb) that binds to the Transferrin Receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) targeting DMPK mRNA. This allows del-desiran to address the underlying cause of disease by reducing DMPK mRNA, releasing MBNL as shown in MARINA participants where del-desiran treatment led to dose-dependent increases in inferred MBNL and, potentially alleviating the spectrum of symptoms that people with myotonic dystrophy type 1 experience.

Myotonic Dystrophy Companies

AMO Pharma Limited, Lupin Ltd., Harmony Biosciences, LLC, Avidity Biosciences, Inc. and others.

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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. However, the symptoms of myotonic dystrophy type 2 are usually milder than those of myotonic dystrophy type 1.

Scope of the Myotonic Dystrophy Market Report

* Coverage- 7MM
* Myotonic Dystrophy Companies- AMO Pharma Limited, Lupin Ltd., Harmony Biosciences, LLC, Avidity Biosciences, Inc., and others.
* Myotonic Dystrophy Therapies- Mexiletine, AOC 1001 (del-desiran), Tideglusib, ATX-01, VX-670, and others.
* Myotonic Dystrophy Market Dynamics: Myotonic Dystrophy Market drivers and Myotonic Dystrophy Market Barriers
* Myotonic Dystrophy Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, 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. Executive Summary of Myotonic Dystrophy

5. Key events

6. Disease background and overview

7. Epidemiology and Patient Population

8. Patient Journey

9. Emerging Therapies

10. Myotonic Dystrophy: 7 Major Market Analysis

11. SWOT Analysis

12. KOL Views

13. Unmet Need

14. Market Access and Reimbursement

15. Appendix

16. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer

About Us

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