Duchenne Muscular Dystrophy Treatment Market: Introduction
- Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive degeneration of the muscles and weakness in them. It is a rare muscle disease, which largely affects men.
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- Duchenne muscular dystrophy (DMD) therapeutics help treat this genetic disorder caused due to absence of dystrophin, a protein in the human body. Symptoms of the disease include weakness in the muscles of the hips, the thighs, the pelvic area, and the shoulders. Later, weakness is observed in the muscles of the arms, the legs, and the trunk. Various therapies and drugs to control DMD are available, which include mutation suppression, exon skipping, and steroid therapy.
- DMD is caused by changes (mutations) of the DMD gene on the X chromosome
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Key Drivers and Restraints of Global Duchenne Muscular Dystrophy Treatment Market
- Increase in the number of patients with Duchenne muscular dystrophy is expected to propel the global Duchenne muscular dystrophy treatment market during the forecast period. According to the National Organization for Rare Disorders, one in 3,500 male babies born worldwide are affected with Duchenne muscular dystrophy. Overall, muscular dystrophies affect 250,000 individuals in the U.S.
- Increase in the number of clinical trials for testing the future treatment for Duchenne muscular dystrophy and wide product pipeline are likely to drive the global market for Duchenne muscular dystrophy treatment during the forecast period. Ataluren, casimersen, golodirsen, suvodirsen, and viltolarsen are five drug molecules undergoing Phase-3 clinical trial for their use in the treatment of Duchenne muscular dystrophy. EXONDYS 51 has been recently approved for treatment of the disease.
- Low rate of treatment caused by delayed diagnosis of the disease is restraining global Duchenne muscular dystrophy treatment market
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Exon Skipping Segment to Witness Significant Growth
- Based on therapeutics, the global Duchenne muscular dystrophy treatment market can be segmented into mutation suppression, exon skipping, and steroid therapy
- The exon skipping segment accounted for the maximum share of the global Duchenne muscular dystrophy treatment market in 2018. The segment is expected to continue its dominance in the next few years, as a majority of exon skipping drugs are undergoing Phase-3 clinical trials. Only one drug approved for Duchenne muscular dystrophy i.e. Exondy 51 falls under the exon skipping therapeutics.
Hospital Pharmacies Segment to Witness Significant Growth
- In terms of distribution channel, the global Duchenne muscular dystrophy treatment market can be divided into hospital pharmacies, retail pharmacies, and online sales
- The hospital pharmacies segment generated the maximum revenues in 2018 and it is anticipated to witness significant growth during the forecast period. This can be attributed to growing number of patients with Duchenne muscular dystrophy being hospitalized. Hence, drugs for Duchenne muscular dystrophy are being sold on a large scale through hospital pharmacies.
North America to Dominate Global Duchenne Muscular Dystrophy Treatment Market
- In terms of region, the global Duchenne muscular dystrophy treatment market can be categorized into: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America dominated the global Duchenne muscular dystrophy treatment market in 2018 and it is expected to maintain its leading position during the forecast period. The dominance of the region can be attributed to increasing research and development activities by major market players, expansion of product offerings by these players, and rise in the demand for early diagnosis in the region.
- The market in Asia Pacific is projected to register the maximum growth rate during the forecast period, owing to increase in the adoption of new products and rise in the incidence of Duchenne muscular dystrophy in the region
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Key Manufacturers Operating in Global Market
The global Duchenne muscular dystrophy treatment market was highly fragmented in 2018. Key manufacturers operating in the global market are:
- Sarepta Therapeutics, Inc.
- Santhera Pharmaceuticals
- PTC Therapeutics
- NS Pharma, Inc.
- Wave Life Sciences
- Daiichi Sankyo Company
- Pfizer Inc.
- Solid Biosciences
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