Myotonic Dystrophy - Pipeline Insight, 2025
Description
DelveInsight’s, “Myotonic Dystrophy - Pipeline Insight, 2025” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs in Myotonic Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Myotonic Dystrophy: Overview
Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood. There are two major forms recognized based on clinical and molecular presentation: Myotonic dystrophy type I (DM1), known as Steinert disease, and myotonic dystrophy type II (DM2), or proximal myotonic myopathy which is a milder variety of DMI. These are progressive, multisystem genetic disorders.
Clinical presentation is diverse and can range from asymptomatic electrical myotonia to severe weakness and disability, including cardiac conduction defects, infertility, cataracts, and insulin resistance. A congenital form of DM type 1 is associated with an apparent developmental disability.
Myotonia is characterized by impaired relaxation of muscles after voluntary contraction due to repetitive depolarization of the muscle membrane. Myotonia, due to myotonic dystrophy, improves with repeated exercise and is worsened by exposure to cold. In 1992, the mutation responsible for DM1 was identified as a CTG expansion located in the 3′ untranslated region of the dystrophia myotonica-protein kinase gene (DMPK).
The pathophysiology of DMI is related to the number of CTG repeats, with more repeats correlating with more severe disease. Those with less than 35 repeats are considered normal, and those that manifest with clinical symptoms typically have greater than 50. Maternal transmissions will often result in greater CTG expansion. Additionally, the newly expanded alleles have a bias for further expansions that leads to the genetic phenomenon of anticipation when transmitted from one generation to the next.
MDs are genetic disorders caused by localised mutations of DNA. Such DNA mutations result in a lack of dystrophin protein in DMD and an alteration of the protein production in DMs. No curative therapies are available to treat the pathogenic causes, and the identification of new therapeutic approaches to target genetic mutations is urgently needed. The most recent strategies to counteract MDs concern gene therapy and repurposing of drugs. Maximizing health and functional independence is the goal of supportive management. This is geared towards monitoring and treating all the medical issues associated with DM. Physical and occupational therapy is recommended for strengthening weakened muscles, evaluation for orthotics, and durable medical equipment needs. Speech-language pathology (SLP) is required for dysphagia and swallowing studies or dysarthria as indicated. SLP is also utilized for intellectual disabilities and learning strategies.
""Myotonic Dystrophy- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myotonic Dystrophy pipeline landscape is provided which includes the disease overview and Myotonic Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Myotonic Dystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Myotonic Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Myotonic Dystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II/III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Myotonic Dystrophy Emerging Drugs
Further product details are provided in the report……..
Myotonic Dystrophy: Therapeutic Assessment
This segment of the report provides insights about the different Myotonic Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:
Myotonic Dystrophy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myotonic Dystrophy therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Myotonic Dystrophy drugs.
Myotonic Dystrophy Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Myotonic Dystrophy: Overview
Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood. There are two major forms recognized based on clinical and molecular presentation: Myotonic dystrophy type I (DM1), known as Steinert disease, and myotonic dystrophy type II (DM2), or proximal myotonic myopathy which is a milder variety of DMI. These are progressive, multisystem genetic disorders.
Clinical presentation is diverse and can range from asymptomatic electrical myotonia to severe weakness and disability, including cardiac conduction defects, infertility, cataracts, and insulin resistance. A congenital form of DM type 1 is associated with an apparent developmental disability.
Myotonia is characterized by impaired relaxation of muscles after voluntary contraction due to repetitive depolarization of the muscle membrane. Myotonia, due to myotonic dystrophy, improves with repeated exercise and is worsened by exposure to cold. In 1992, the mutation responsible for DM1 was identified as a CTG expansion located in the 3′ untranslated region of the dystrophia myotonica-protein kinase gene (DMPK).
The pathophysiology of DMI is related to the number of CTG repeats, with more repeats correlating with more severe disease. Those with less than 35 repeats are considered normal, and those that manifest with clinical symptoms typically have greater than 50. Maternal transmissions will often result in greater CTG expansion. Additionally, the newly expanded alleles have a bias for further expansions that leads to the genetic phenomenon of anticipation when transmitted from one generation to the next.
MDs are genetic disorders caused by localised mutations of DNA. Such DNA mutations result in a lack of dystrophin protein in DMD and an alteration of the protein production in DMs. No curative therapies are available to treat the pathogenic causes, and the identification of new therapeutic approaches to target genetic mutations is urgently needed. The most recent strategies to counteract MDs concern gene therapy and repurposing of drugs. Maximizing health and functional independence is the goal of supportive management. This is geared towards monitoring and treating all the medical issues associated with DM. Physical and occupational therapy is recommended for strengthening weakened muscles, evaluation for orthotics, and durable medical equipment needs. Speech-language pathology (SLP) is required for dysphagia and swallowing studies or dysarthria as indicated. SLP is also utilized for intellectual disabilities and learning strategies.
""Myotonic Dystrophy- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myotonic Dystrophy pipeline landscape is provided which includes the disease overview and Myotonic Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Myotonic Dystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Myotonic Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Myotonic Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve Myotonic Dystrophy.
This segment of the Myotonic Dystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II/III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Myotonic Dystrophy Emerging Drugs
- Mexiletine: Lupin
- Pitolisant: Harmony Biosciences
- ARO-DM1: Arrowhead Pharmaceuticals, Inc.
Further product details are provided in the report……..
Myotonic Dystrophy: Therapeutic Assessment
This segment of the report provides insights about the different Myotonic Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Myotonic Dystrophy
- There are approx. 20+ key companies which are developing the therapies for Myotonic Dystrophy. The companies which have their Myotonic Dystrophy drug candidates in the most advanced stage, i.e. Phase III include, Lupin.
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
- Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
- Product Type
Myotonic Dystrophy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myotonic Dystrophy therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Myotonic Dystrophy drugs.
Myotonic Dystrophy Report Insights
- Myotonic Dystrophy Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Myotonic Dystrophy drugs?
- How many Myotonic Dystrophy drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Myotonic Dystrophy?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Myotonic Dystrophy therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Myotonic Dystrophy and their status?
- What are the key designations that have been granted to the emerging drugs?
- AMO Pharma
- Lupin
- Harmony Biosciences
- Avidity Biosciences
- Dyne Therapeutics
- Vertex Pharmaceuticals
- Arrowhead Pharmaceuticals, Inc.
- Pepgen Corporation
- Aparito
- Tideglusib
- Mexiletine
- Pitolisant
- AOC 1001
- DYNE-101
- VX-670
- ARO-DM1
- PGN EDODM1
- IONIS-877864
Table of Contents
80 Pages
- Introduction
- Executive Summary
- Myotonic Dystrophy: Overview
- Introduction
- Causes
- Pathophysiology
- Signs and Symptoms
- Diagnosis
- Treatment
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Myotonic Dystrophy– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Mexiletine: Lupin
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Pitolisant: Harmony Biosciences
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- ARO-DM1: Arrowhead Pharmaceuticals, Inc.
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Myotonic Dystrophy Key Companies
- Myotonic Dystrophy Key Products
- Myotonic Dystrophy- Unmet Needs
- Myotonic Dystrophy- Market Drivers and Barriers
- Myotonic Dystrophy- Future Perspectives and Conclusion
- Myotonic Dystrophy Analyst Views
- Myotonic Dystrophy Key Companies
- Appendix
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