Myotonic Dystrophy Treatment Market is projected to witness substantial growth over the next few years, driven by advancements in treatment modalities and increasing awareness of the condition. The pipeline for Myotonic Dystrophy Therapeutics includes promising candidates that aim to not only treat acute attacks but also prevent recurrence.
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 September 2024:- 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. The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks. Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.
- Based on DelveInsight's estimates, in 2023, the diagnosed prevalent population of Myotonic Dystrophy in the United States was estimated to be approximately 54 thousand.
- As per the DelveInsight analysis, it was observed that Japan accounted for approximately 12% diagnosed prevalent population of Myotonic Dystrophy among 7MM, in the year 2023.
- According to DelveInsight estimates, in 2023 in the US, there were ~541 cases of Congenital form, ~3 thousand cases of Infantile form, ~1 thousand cases of Juvenile form, ~39 thousand cases of Adult-onset form, and around ~8 thousand cases of Late-onset form.
- 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
Myotonic Dystrophy Market Insights
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. DM is of two types; DM type 1 is caused by the expansion of a CTG triplet repeat in DMPK, whereas DM type 2 is caused by the expansion of a CCTG tetramer repeat in CNBP. However, the symptoms of DM2 are usually milder than those of DM1.
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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 Market Outlook
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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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
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