DelveInsight’s “Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Myotonic Dystrophy market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Myotonic Dystrophy market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Myotonic Dystrophy: An Overview
There are two main types of myotonic dystrophy (DM) — DM type 1 (DM1) and DM type 2 (DM2). DM1 is also known as Steinert disease, named after Dr Steinert, who, along with colleagues, first described the classic form in the medical literature in 1909. DM2 is also known as Ricker syndrome or proximal DM, or PROMM. DM1 is further classified into congenital DM1, childhood DM1, adulthood DM1, and late-onset DM1.
DM1 and DM2 are caused by an expansion of DNA tandem repeats, resulting in an RNA gain of function mutation. DM1 is caused by the expansion of cytosine-thymine-guanine (CTG) repeat in the 3’-untranslated region of the DMPK gene on chromosome 19q13.3. DM2 results from the expansion of cytosine-cytosine-thymine-guanine (CCTG) tetranucleotide located in the intron of the CNB or ZNF9 gene on chromosome 3q21.3. DM1 is estimated to affect about 1 in 8,000–20,000 people in the general population. However, the prevalence of DM1 and DM2 varies greatly among different countries and ethnic groups.
Myotonic Dystrophy Market Key Facts
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In 2021, the Myotonic Dystrophy market size was approximately USD 80 million in the 7MM.
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The total diagnosed prevalent patient population of Myotonic Dystrophy in the 7MM countries was close to 80,000 cases in 2021.
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As per the estimates, the US had the highest patient population of Myotonic Dystrophy in 2021.
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Among the EU5 countries, Germany had the highest diagnosed patient population of Myotonic Dystrophy, with over 9,000 cases, followed by France in 2021. Spain had the lowest diagnosed prevalent patient population of Myotonic Dystrophy, with approximately 6,000 cases in 2021 among the EU5.
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Several companies are working robustly on many new therapies, such as AMO-02 (AMO Pharma Limited), mexiletine (Lupin Ltd.), and pitolisant (Harmony Biosciences, LLC).
The market outlook section of the report helps to build a detailed comprehension of the historical, current and forecasted Myotonic Dystrophy market size by analyzing the impact of current and emerging pipeline therapies. It also thoroughly assesses the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Myotonic Dystrophy market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Myotonic Dystrophy Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Report Covers the Myotonic Dystrophy Epidemiology, Segmented as –
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Total Diagnosed Prevalent Cases of DM [2019–2032]
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Type-specific Diagnosed Cases of DM [2019–2032]
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Age-specific Diagnosed Cases of DM [2019–2032]
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Comorbidity-associated Diagnosed Cases of DM [2019–2032]
Myotonic Dystrophy Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Myotonic Dystrophy market or expected to be launched during the study period. The analysis covers the 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 analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Latest Developments in the Myotonic Dystrophy Therapeutics Market
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In August 2021, AMO Pharma Limited received an innovative licensing and access pathway (ILAP) designation from the UK Medicines and healthcare products regulatory agency (MHRA) for AMO-02 (tideglusib) for the development of treatment of CDM1.
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In November 2020, US FDA granted an RPDD to AMO-02 for the development of the treatment of CDM.
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In June 2020, the US FDA granted ODD to mexiletine hydrochloride to treat DM.
Myotonic Dystrophy Therapeutics Analysis
Several major pharma and biotech companies are developing therapies for Myotonic dystrophy. Currently, Lupin is leading the therapeutic market with its Myotonic dystrophy drug candidates in the most advanced stage of clinical development.
The Leading Players in the Myotonic Dystrophy Therapeutics Market Include:
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AMO Pharma Limited
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Arthex Biotech
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Astellas Gene Therapies
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Avidity Biosciences
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Dyne Therapeutics
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Entrada Therapeutics
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Enzerna
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Harmony Biosciences, LLC
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Locana, Inc.
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Lupin Ltd.
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NeuBase Therapeutics
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Nexien BioPharma
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Pepgen Corporation
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Sangamo Therapeutics
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Syros Pharmaceuticals
And Many Others
Myotonic dystrophy Emerging and Marketed Drugs Covered in the Report Include:
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AMO-02 (tideglusib): AMO Pharma Limited
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AOC 1001: Avidity Biosciences, Inc.
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DYNE-101: Dyne Therapeutics
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Mexiletine: Lupin Ltd.
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Pitolisant: Harmony Biosciences, LLC
And Many More
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Myotonic Dystrophy Competitive Intelligence Analysis
4. Myotonic Dystrophy Market Overview at a Glance
5. Myotonic Dystrophy Disease Background and Overview
6. Myotonic Dystrophy Patient Journey
7. Myotonic Dystrophy Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Myotonic Dystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Myotonic Dystrophy Unmet Needs
10. Key Endpoints of Myotonic Dystrophy Treatment
11. Myotonic Dystrophy Marketed Products
12. Myotonic Dystrophy Emerging Drugs and Latest Therapeutic Advances
13. Myotonic Dystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Myotonic Dystrophy Market Outlook (In US, EU5, and Japan)
16. Myotonic Dystrophy Access and Reimbursement Overview
17. KOL Views on the Myotonic Dystrophy Market
18. Myotonic Dystrophy Market Drivers
19. Myotonic Dystrophy Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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