Duchenne Muscular Dystrophy (DMD) Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018

Duchenne Muscular Dystrophy (DMD) Pipeline Drugs Assessment Overview:

Duchenne muscular dystrophy is a genetic disorder which causes progressive muscle degeneration and weakness, and it is one of the nine types of muscular dystrophy. Duchenne muscular dystrophy mostly affects males, but in rare cases occurs in females. Onset of the disease is generally 3 to 4 years of age and begins on hips, pelvic region, shoulders, and upper legs. Symptoms may include delayed or difficulty inability to stand, walk and difficulty in speaking.
Disease is diagnosed through the patient history, blood test for creatinine phosphokinase levels, molecular genetic testing, and muscle biopsy. There is no cure available for the disease till now. But the medications are available forrelieving the symptoms of the disease. Eteplirsen is used to treat the patients with specific gene mutation of gene that leads to DMD. Deflazacort, approved in 2017, becoming first corticosteroid to treat Duchenne muscular dystrophy.

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Segmentation:
By formulation, Duchenne muscular dystrophy pipeline drugs are segmented into
• Oral
• Parenteral
• Others
By therapy, Duchenne muscular dystrophy pipeline drugs are segmented into
• Exon Skipping
• Gene Therapy
• Corticosteroids
• Others
By trial phase, Duchenne muscular dystrophy pipeline drugs are segmented into
• Phase I
• Phase II
• Phase III
• Phase IV
By company, Duchenne muscular dystrophy pipeline drugs are segmented into
• Pfizer Inc. (U.S.)
• ReveraGenBioPharma, Inc. (U.S.)
• Sarepta Therapeutics Inc. (U.S.)
• Daiichi Sankyo Company (Japan)
• BioMarin Pharmaceutical Inc. (U.S.)
• NS Pharma, Inc. (Japan)
• TAIHO PHARMACEUTICAL CO. (Japan)

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Space Analysis:
Until the approval of exon skipping agent eteplirsen, carticostroids are the only potential treatment for the treatment of duchenne muscular atrophy, but with many side effects. Duchenne muscular dystrophy pipeline has the robust drug molecules and therapies such as gene therapy, exon skipping agents and also promising corticosteroids for the treatment. Furthermore, Catabasis Pharmaceuticals has been developing NF-kB protein inhibitor in phase-II development to treat duchenne muscular dystrophy. Moreover, there are numerous early stage development of drugs including, drugs which targets calcium regulation, fibrosis and oxidative stress.
• In November 2016, Wave Life Sciences Ltd. Initiated the Phase I clinical trial for WVE-210201 Duchenne muscular dystrophy patients amenable to exon 51 skipping.

Report Description:
Duchenne Muscular Dystrophy Pipeline Drugs Assessment report studies the various therapeutics under clinical development for XYX treatment along with targets for various drug candidate. The report provides plethora of information pertaining to trail phases, companies involved in the Duchenne muscular dystrophy pipeline drugs development. This report studies the dynamics of the Duchenne Muscular Dystrophy Pipeline Drugs i.e. drivers, challenges, and opportunities which are significantly impacting the product development. The report provides various information pertaining the clinical trials such as designation, grants, patents, and technology among others. Moreover, the report on Duchenne muscular dystrophy pipeline drugs assessment comprehensively presents the geographic location, trial status information along with key players involved in the therapeutics development.


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Key Features of the Report:
• Provides the information related to universities and research institutes working in the therapeutics development
• Report comprehensively covers the all active and discontinued studies
• Studies the entire pipeline with special emphasis on companies actively involved in the therapeutics development
• Presents the prominent targets for drug development in each stage of clinical trial
• Provides the in-depth analysis on the each drug candidates in the clinical trial phases

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