The Myotonic Dystrophy drugs in development market research report provides 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. GlobalData’s report assesses key aspects of the companies and drugs in development for Myotonic Dystrophy. Buy the report here.
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The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Myotonic Dystrophy and features dormant and discontinued products.
GlobalData tracks 52 drugs in development for Myotonic Dystrophy by 48 companies/universities/institutes. The top development phase for Myotonic Dystrophy is discovery with 28 drugs in that stage. The Myotonic Dystrophy pipeline has 43 drugs in development by companies and nine by universities/ institutes. Some of the companies in the Myotonic Dystrophy pipeline products market are: University of Florida, Vertex Pharmaceuticals and Enzerna Biosciences.
The key targets in the Myotonic Dystrophy pipeline products market include Myotonin Protein Kinase, RNA, and Voltage Gated Sodium Channel (SCN).
The key mechanisms of action in the Myotonic Dystrophy pipeline product include Myotonin Protein Kinase Inhibitor with 13 drugs in Phase III. The Myotonic Dystrophy pipeline products include five routes of administration with the top ROA being Oral and nine key molecule types in the Myotonic Dystrophy pipeline products market including Small Molecule, and Gene Therapy.
Myotonic Dystrophy overview
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.
For a complete picture of Myotonic Dystrophy’s pipeline drug market, buy the report here.
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GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.
