The global myotonic dystrophy drug market is anticipated to grow at a significant CAGR during the forecast period (2022-2028). Myotonic dystrophy is a muscular dystrophy caused by a long-term genetic mutation. It is the most common type of muscular dystrophy in adulthood, affecting roughly one out of every 8,000 people. Muscle atrophy and weakness worsen over time as a symptom. Muscles contract frequently and take a long time to relax. Some people have a relatively mild type, while others have a severe one that affects them from birth. This is owing to the condition’s molecular foundation, which is caused by a dynamic mutation called a triplet repeat expansion, which occurs when three base pairs of DNA are present in varied copies numbers.
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The primary factor propelling the growth of global myotonic dystrophy market is the introduction of novel disease therapeutics and increased R&D activities. For instance, in November 2021, Locanabio, Inc., a genetic pharmaceuticals business focused on developing therapies for patients with severe neuromuscular, neurodegenerative, and retinal illnesses, announced new preclinical data from its myotonic dystrophy type 1 (DM1) program. Locanabio scientists used their CORRECTx platform to show a dose-dependent reduction in toxic CUG foci in both DM1 patient muscle cells and a preclinical mouse model of DM1, which resulted in an alternative RNA splicing correction and a statistically significant reduction in myotonia, or muscle weakness. Further, in April 2020, Scientists from the University’s Schools of Life Sciences and Chemistry have discovered that inhibiting a molecule in patients’ cells called CDK12 can potentially develop a therapy to alleviate some of the symptoms and support treat this incurable condition.
Some major key players in the market include Isis Pharmaceuticals, Inc., and Lupin Ltd., among others. The market players are contributing significantly to the growth of the market by the adoption of various strategies including mergers & acquisitions, geographical expansion, partnerships and collaborations, and new product launches, to stay competitive in the market. For instance, in October 2021, AOC 1001 has been granted Fast Track designation by the Food and Drug Administration (FDA) for the treatment of myotonic dystrophy type 1 (DM1). AOC 1001 is a monoclonal antibody that binds to transferrin receptor 1 and is coupled to a short interfering RNA that targets DMPK mRNA. AOC 1001 is expected to address the fundamental cause of DM1 by lowering DMPK mRNA levels in skeletal, cardiac, and smooth muscle. Data from preclinical tests back up the designation, showing that AOC 1001 successfully transported the small interfering RNA to muscle cells, resulting in dose-dependent decreases in DMPK mRNA.
Market Coverage
- The market number available for – 2021-2028
- Base year- 2021
- Forecast period- 2022-2028
Segment Covered-
- By Type
- By Application
Regions Covered-
- North America
- Europe
- Asia-Pacific
- Rest of the World
Competitive Landscape– Isis Pharmaceuticals, Inc., and Lupin Ltd., among others.
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Global Myotonic Dystrophy Drug Market Report by Segment
By Type
- Myotonic Dystrophy Type 1 (DM1)
- Myotonic Dystrophy Type 2 (DM2)
By Application
- Hospital
- Clinic
- Others
Global Myotonic Dystrophy Drug Market Report by Region
North America
- United States
- Canada
Europe
- UK
- Germany
- Spain
- France
- Italy
- Rest of Europe
Asia-Pacific
- India
- China
- Japan
- South Korea
- Rest of APAC
Rest of the World
- Latin America
- Middle East & Africa
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