Myotonic Dystrophy – Drugs In Development, 2024
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Myotonic dystrophy (DM), also known as dystrophia myotonica, is a group of genetic disorders characterized by progressive muscle weakness, myotonia (delayed relaxation of muscles after contraction), and various multi-systemic manifestations. It is one of the most common forms of adult-onset muscular dystrophy and can affect multiple organs and systems in the body.
There are two main types of myotonic dystrophy, DM1 and DM2. DM1, also called Steinert’s disease, is the most common form; it typically presents in adolescence or adulthood and can vary widely in severity. Symptoms may include muscle weakness, myotonia, cataracts, heart abnormalities, hormonal disturbances, and intellectual impairment in some cases. DM2 is less common and usually milder than DM1. Symptoms are similar but tend to appear later in life and progress more slowly. It may present with muscle weakness, myotonia, and, in some cases, heart rhythm abnormalities. Myotonic dystrophy is caused by genetic mutations in specific genes: DM1 is caused by an expanded repetition of CTG nucleotide sequences in the DMPK gene on chromosome 19, and DM2 is caused by an expanded repetition of CCTG nucleotide sequences in the CNBP gene on chromosome 3. Symptoms of myotonic dystrophy involve various body systems and may include muscle weakness, particularly in facial, neck, and distal limb muscles; myotonia, causing difficulty in relaxing muscles after contraction, leading to stiffness or difficulty with movements; eye abnormalities such as cataracts or retinal abnormalities; cardiac issues, including arrhythmias, cardiomyopathy, or conduction abnormalities; endocrine abnormalities like insulin resistance, thyroid issues, and gonadal dysfunction; and cognitive impairment, particularly in DM1, which can manifest as learning difficulties, cognitive decline, or behavioral changes.
The Myotonic Dystrophy drugs in development market research report provide comprehensive information on the therapeutics under development for Myotonic Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Myotonic Dystrophy and features dormant and discontinued products.
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Scope
- Therapeutics in Development: Covering 50 molecules, with 42 developed by companies and the rest by universities/institutes.
- Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.
- Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.
- Development History: Access the complete research and development history.
- Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.
Reasons to Buy
- Holistic insights: Understand the broad spectrum of Myotonic Dystrophy therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
- Pipeline Exploration: Explore the detailed Myotonic Dystrophy pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.
- Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Myotonic Dystrophy treatments for adaptable decision-making.
- Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.
Key Players
AMO Pharma LtdAnima Biotech Inc
ARTHEx Biotech SL
Astellas Gene Therapies
Avidity Biosciences Inc
Base4 Biotechnology Inc
CRISPR Therapeutics AG
Denali Therapeutics Inc
Design Therapeutics Inc
DR.Noah Biotech Inc
Dyne Therapeutics Inc
EditForce Inc
Entrada Therapeutics Inc
Enzerna Biosciences LLC
Expansion Therapeutics Inc
Faze medicines
Fulcrum Therapeutics Inc
Generian Pharmaceuticals Inc
Harmony Biosciences Holdings Inc
Healx Ltd
Juvena Therapeutics Inc
Kate Therapeutics Inc
Locanabio Inc
Loqus23 Therapeutics Ltd
Lupin Ltd
Myogem Health Company SL
Nexien Biopharma Inc
Osaka University
Osaka University Hospital
Pepgen Inc
Pfizer Inc
Prime Medicine Inc
Scriptr Global Inc
Syros Pharmaceuticals Inc
The Scripps Research Institute
The University of Nottingham
Triplet Therapeutics Inc
University of Florida
University of Illinois
University of Rochester Medical Center
Vertex Pharmaceuticals Inc
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