Market Research Report on Muscular Dystrophies

Muscular dystrophies (MD) are a spectrum of genetic disorders characterized by muscle weakness that, which in severe forms of the disease can lead to loss of gait and premature...

Muscular dystrophies (MD) are a spectrum of genetic disorders characterized by muscle weakness that, which in severe forms of the disease can lead to loss of gait and premature death. Doctors share common histological features, such as reduced muscle fiber size, degeneration, and the presence of connective tissue and adipose tissue instead of muscle. Duchenne muscular dystrophy (DMD) is the most common subtype in the children, while myotonic dystrophy is more common in adults.

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Ataluren targets a subset of patients with DMD and has been available in selected markets after its European approval in 2014, eteplirsen (Sarepta’s Exondys 51) received conditional approval in the United States in 2016 and targets a different group of patients with DMD who share a genetic mutation similar to the basis of their disease. Although both drugs target the underlying cause of the disease and, therefore, could alter its course, the efficacy observed so far in clinical trials seems modest and the unmet need for effective therapies that modify the disease.

The unmet need for effective treatments capable of significantly delaying or stopping progressive muscle degeneration in DMD, as well as in other severe and moderate cases is high. It is expected that the growing research and development, the introduction of new therapies, the growing demand for effective therapies in patients and an increase in the prevalence of the disease will drive the growth of the therapeutics for muscular dystrophy market. According to the Centers for Disease Control and Prevention, in 2016, the prevalence of Duchenne muscular dystrophy and Becker muscular dystrophy (DBMD) was 1 in 7,250 men aged 5 to 24 years. The increasing prevalence of chronic diseases such as cardiovascular, neurovascular and arthritis is another supporting factor for the growth of this disease. According to the World Health Organization, cardiovascular diseases caused 17.7 million deaths in 2015, representing 31% of all deaths worldwide. However, stricter regulations for product approval and high product costs may limit the market expansion.

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North America accounts for the largest base of sufferers with this ailment. Innovation in new products and government awareness programs will increase the awareness of people in North America about the issues related with the disease. Muscular Dystrophy of United Parent Projects has launched the Duchenne World Awareness Day on September 7 that aims to raise awareness about the disease and its consequences. Europe is the second largest market for muscular dystrophies. The Asia-Pacific is expected to grow faster due to rapid population growth and the increasing prevalence of chronic diseases. Increased health care coverage and increased investment in research and development along with the growing awareness of the population and greater coverage of public and private healthcare will create huge opportunities for the pharmaceutical companies to develop effective drugs for treatment.

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AMO Pharma, Akashi Therapeutics, aTyr Pharma, Catabasis Pharmaceuticals, Biogen, FibroGen, EspeRare Foundation, Milo Biotechnology, Italfarmaco SpA, PTC Therapeutics, Pfizer, Roche, ReveraGen BioPharma, Sarepta Therapeutics, and Santhera Pharmaceuticals are the major players of the market.

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