Duchenne muscular dystrophy (DMD) is one of the most common types of muscular dystrophy, which is an X-linked inherited genetic disorder characterized by gradual skeletal muscle weakness. According to NHS, in every year 100 boys are born with Duchenne muscular dystrophy in the U.K. As per DMD surveillance report by Center for Disease Control and Prevention (CDC), 349 new cases of Duchenne muscular dystrophy were reported in 2010 in the U.S. Duchenne muscular dystrophy usually affects male child and first signs and symptoms of the disease are observed at the age of 2-3 years. Duchenne muscular dystrophy worsens quickly which results in loss of ability to walk and eventually causes death within 20-30 years of age. Currently there is no curative treatment for Duchenne muscular dystrophy however symptomatic treatment approaches such as pain alleviating drugs and steroids to slow down the progression of disease are recommended by majority of physicians. Treatment is mainly aimed to maximize the quality of life.
Continuous research on gene therapy, exon skipping drugs and stem cell therapy for the treatment of Duchenne muscular dystrophy is expected to fuel the global market for Duchenne muscular dystrophy treatment market over the forecast period. BioMarin Pharmaceutical Inc. is developing exon skipping drug Drisapersen which is currently being evaluated in phase III clinical trial. In 2014, PTC Therapeutics International Limited obtained orphan drug designation and conditional approval for Translarna–first ever gene therapy for treatment of Duchenne muscular dystrophy, in Europe. However, low treatment seeking rate due to delayed diagnosis and fast prognosis of a disorder is the major factor limiting the growth of global Duchenne muscular dystrophy treatment market.
Specific guidance for drug development for Duchenne muscular dystrophy drugs published by the U.S. FDA in 2015 is expected to accelerate the new drug development for treatment of a disorder. Moreover, new approaches such as utrophin modulation approach for the treatment of Duchenne muscular dystrophy are expected to launch in the market during the forecast period. For instance, Summit Therapeutics plc. is evaluating ezutromid in phase II clinical trials which has received orphan drug designation and fast track approval from U.S. FDA.
Collaborations between key players involved in development of drugs for Duchenne muscular dystrophy would increase the pace of new drug development for the disease. By product type, the global market for Duchenne muscular dystrophy treatment has been classified into pain management drugs and corticosteroids. Corticosteroids product type segment is expected to hold maximum share in global Duchenne muscular dystrophy treatment market.
Based on end user, the global Duchenne muscular dystrophy treatment market has been classified as hospitals, clinics and home care settings. Hospitals end user segment is expected to hold maximum share in the global market owing to increasing rate of hospitalization for DMD.
On the basis of regional presence, global Duchenne muscular dystrophy treatment market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America will continue to dominate the global Duchenne muscular dystrophy treatment market due to high prevalence of the disease. Europe is expected to hold second largest market share in global market. Increasing R&D activities in the DMD area is expected to bring huge growth of global market during the forecast period.
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Some of the major players operating in global Duchenne muscular dystrophy treatment market are PTC Therapeutics, Summit Therapeutics Plc., Sarepta Therapeutics, BioMarin Pharmaceutical Inc., Pfizer Inc., (Pharmacia & Upjohn LLC) and others. Companies are involved in collaboration agreements for R&D in order to exploit maximum potential.
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