Muscular Dystrophy is characterized by a collection of muscle-wasting conditions. The disorder is caused by genetic mutations which interfere with the production of muscle protein dystrophin, necessary to build muscles. Muscular dystrophy is a genetic disease, consequently, the chance of an individual developing a disease increases with a history of muscular dystrophy in the family. The symptoms include shortening of muscles and tendons, the curvature of spine, weakening of heart muscles leading to cardiac problems, and breathing problems. The most common forms of muscular dystrophies include duchenne muscular dystrophy, becker muscular dystrophy, myotonic disease, congenital disease and oculopharyngeal muscular dystrophy. The cure for any form of muscular dystrophy is currently unspecified, but the symptoms can be relived through physical therapy, exercise, rehabilitative devices like a powered wheelchair, respiratory care and surgery.
The common muscular dystrophy types:
Most common form of muscular dystrophy in children caused by lack of protein dystrophin. The onset of symptoms is seen at the age of 3, generally wheelchair-bound by 12. The Curvature in spine, heart, and lungs effects are the symptoms. Duchenne muscular dystrophy (DMD) is an X-linked disease predominantly affecting males, resulting in uniform muscle wasting. Death occurs due to respiratory failure by mid-twenties.
Similar to Duchenne, however, with later onset and slow progression of the illness. Death occurs in the mid-forties.
Initially affect eyelids, throat, and face followed by pelvis and shoulder. The onset of illness is seen between the ages 40 and 50.
Most common form of muscular dystrophy in adults also called as Steinert's disease. Characterized by the inability of muscles to relax after contraction. Cataracts, insomnia, and arrhythmia are the symptoms.
The disease prevails from birth or before the age of 2. Significant impairment is caused by a swift progression of the disease. Congenital muscular dystrophy (CMD) causes learning disabilities and mental retardation.
Rising prevalence of muscular dystrophies with unspecific current medications is expected to fuel the muscular dystrophies treatment market
According to the research conducted by the Karger Journal on 2014, the global prevalence for muscular dystrophies accounted between 19.8 and 25.1 per 100,000 person every year with myotonic dystrophy (0.5-18.1 per 100,000), duchenne muscular dystrophy (1.7-4.2) and facioscapulohumeral muscular dystrophy (3.2-4.6 per 100,000) to be the most common types of disorders. The initial research phase of the therapies and ongoing clinical trials for the development of innovative products drives the global muscular dystrophy treatment market. Also, specific guidance for drug development for muscular dystrophy drugs published by the U.S. FDA in 2015 is expected to accelerate the new drug development for the treatment of a disorder. The emergence of disease-modifying therapies as a replacement for the unmet medical needs of patients who are not amenable to mutation-specific drugs is likely expected to fuel the revenue of the global muscular dystrophy treatment market in the forecast period. However, the fast prognosis of a disorder and low treatment seeking rate due to delayed diagnosis are the major limiting factors for the growth of global muscular dystrophy treatment market.
Molecular therapy segment is expected to generate larger revenue with higher efficiency in clinical trials
The global muscular dystrophy treatment market is segmented on the basis of treatment, diagnosis, end user and geography.
On the basis of treatment, the global muscular dystrophy treatment market is segmented into:
On the basis of diagnosis, the global muscular dystrophy treatment market is segmented into:
On the basis of end user, the global muscular dystrophy treatment market is segmented into:
Specific guidance by FDA is expected to dominate North America the global market
Regional segmentation of the global muscular dystrophy treatment market by Coherent Market Insights comprises North America, Latin America, Europe, Asia-Pacific and The Middle East and Africa. According to the Centers for Disease Control and Prevention estimates in 2007, 349 out of 2.37 million males aged 5 to 24 years were reported to have duchenne muscular dystrophy or becker muscular dystrophy in the United States. North America is expected to dominate the global muscular dystrophy treatment market owing to the absence of marketed pharmacological therapies indicated to treat muscular dystrophy, rising prevalence of the disease and rising research and development for producing effective drugs. Europe is expected to boost the market share in the forecast period with the growing public awareness and increasing research activities.
Launch of efficient corticosteroids to rise the market demand
The key players operating the global muscular dystrophy treatment market include Santhera Pharmaceuticals, PTC Therapeutics, BioMarin Pharmaceutical, Sarepta Therapeutics and Eli Lilly. The approval and launch of oral corticosteroid Emflaza in 2017, has been found highly effective in patients to regain strength and restore their ability to walk. The other vendors include Acceleron Pharma, Capricor Therapeutics, Bristol-Myers Squibb, Lexicon Pharmaceuticals, and Pfizer.
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About Author
Ghanshyam Shrivastava - With over 20 years of experience in the management consulting and research, Ghanshyam Shrivastava serves as a Principal Consultant, bringing extensive expertise in biologics and biosimilars. His primary expertise lies in areas such as market entry and expansion strategy, competitive intelligence, and strategic transformation across diversified portfolio of various drugs used for different therapeutic category and APIs. He excels at identifying key challenges faced by clients and providing robust solutions to enhance their strategic decision-making capabilities. His comprehensive understanding of the market ensures valuable contributions to research reports and business decisions.
Ghanshyam is a sought-after speaker at industry conferences and contributes to various publications on pharma industry.
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