Myotonic Dystrophy Market Size in the 7MM was ~USD 80 Million in 2023, estimated DelveInsight

Myotonic Dystrophy Market

DELHI, DELHI, INDIA, June 23, 2024 /EINPresswire.com/ -- DelveInsight’s ‘Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034’ report delivers an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of DM in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Key Takeaways from the Myotonic Dystrophy Market Report
• June 2024:- Avidity Biosciences Inc.- A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1. A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
• June 2024:- ARTHEx Biotech S.L.- A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1. The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
• June 2024:- Vertex Pharmaceuticals Incorporated- A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1. The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
• Based on DelveInsight's assessment in 2023, the 7MM had 88,322 diagnosed prevalent cases of Myotonic Dystrophy, and it is projected that these cases will experience an increasing growth trend with an estimated CAGR during the forecast period (2024−2034).
• According to DelveInsight's analysis, in the US in 2023, diagnosed prevalent cases of Myotonic Dystrophy were reported as 2,625 for children (<18 years) and 30,184 for adults (>18 years).
• According to DelveInsight’s estimates, among the type-specific diagnosed prevalent cases of Myotonic Dystrophy in the US, were more prevalent for DM1.
• The leading Myotonic Dystrophy Companies such as Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, 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 Diagnosed Prevalent Cases
• Age-specific Diagnosed Prevalent Cases
• Type-specific Diagnosed Prevalent Cases
• Type-specific Diagnosed Prevalent Cases of DM1
• Comorbidity-associated Diagnosed Cases

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Myotonic Dystrophy Market Insights
Myotonic Dystrophy is a rare, inherited disorder with multi-systemic effects that impact the skeletal muscles, eyes, heart, skin, gastrointestinal, endocrine, respiratory, and central nervous systems. Despite several pre-clinical developments, no specific disease-modifying therapy is currently available. Management consists primarily of monitoring for complications and standard of care (assistive devices, hormone therapy, and pain medication). A few clinical studies have systematically evaluated the use of therapeutic agents in DM. The lack of scientific evidence paired with the multi-systematic and highly variable presentation of the disease makes identifying and selecting appropriate medications particularly challenging for prescribing physicians.

Myotonic Dystrophy Drug Market
According to DelveInsight's’ estimates, the Myotonic Dystrophy market in 7MM is expected to show positive growth, during the forecast period (2024–2034), mainly attributed to the anticipated launch of emerging therapies and the increasing cases of Myotonic Dystrophy. The Myotonic Dystrophy pipeline features several drugs in mid- to late-stage development poised for future approval. Anticipated launches of emerging therapies from companies like AMO Pharma Limited, Harmony Biosciences, Vertex Pharmaceuticals, among others, are expected to positively impact the market. Notably, EditForce, Inc. is advancing EF-210, a novel gene therapy program utilizing its proprietary PPR platform technology, targeting Myotonic Dystrophy Type 1 (DM1). EF-210 is currently undergoing preclinical development, promising innovative prospects in DM1 treatment with potential advancements in gene therapy approaches.

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Myotonic Dystrophy Emerging Drugs
Myotonic Dystrophy drug developers are gradually shifting their attention toward Myotonic Dystrophy to meet the patient pool’s current demands and counter the unmet needs of the therapeutic market. Several companies are working robustly on many new therapies, such as AMO-02 (AMO Pharma Limited), mexiletine (Lupin Ltd.), and pitolisant (Harmony Biosciences, LLC). AMO-02 (AMO Pharma Limited) is in development for treating congenital myotonic dystrophy (CDM) and has the potential for use in additional CNS, neuromuscular and other orphan indications. It has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. Currently, the company conducts two Phase II/III studies in children and adolescents with CDM.

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Scope of the Myotonic Dystrophy Market Report
• Coverage- 7MM
• Myotonic Dystrophy Companies- Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, 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. Disease background and overview
6. Epidemiology and Patient Population
7. Patient Journey
8. Emerging Therapies
9. Other Assets
10. Myotonic Dystrophy (DM): 7MM Analysis
11. SWOT Analysis
12. KOL Views
13. Unmet Need
14. Market Access
15. Acronyms and Abbreviations
16. Appendix
17. DelveInsight Capabilities
18. Disclaimer
19. 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.

Yash Bhardwaj
DelveInsight
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