Muscular Dystrophy Disease Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018
Muscular Dystrophy Disease Pipeline Drugs Assessment
Muscular Dystrophy is a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of skeletal muscles that control movement. This damage and weakness is because of the lack of protein known as dystrophin which is necessary for muscle function. Muscular dystrophy can occur at any age but most diagnosis occurs in childhood. Prognosis of Muscular Dystrophy depends on the type and severity of symptoms but most of the individuals with muscular dystrophy do lose the ability to walk. 30 different types of MD will have different types of symptoms, most common symptoms are trouble in walking, difficulty in swallowing, muscle cramps and weakness etc.
Muscular Dystrophy can be diagnosed by blood tests, electromyography, muscle biopsy etc. Muscular Dystrophy treatment may include physical therapy, respiratory therapy, speech therapy, orthopedic appliances used for support, and corrective orthopedic surgery. Drug therapy includes corticosteroids to slow muscle degeneration, anticonvulsants to control seizures and some muscle activity, immune asuppressants to delay some damage to dying muscle cells, and antibiotics to fight respiratory infections
By Trial Phase, Muscular Dystrophy pipeline drugs are segmented as:
- Preclinical Trials
- Phase 1
- Phase 2
- Phase 3
- Phase 4
By Company, Muscular Dystrophy pipeline drugs are segmented as:
- NS Pharma, Inc
- ReveraGenBioPharma, Inc.
- Sarepta Therapeutics
- Acceleron Pharma, Inc.
- PTC Therapeutics
By Drugs, Muscular Dystrophy pipeline drugs are segmented as:
By Route of Administration, Muscular Dystrophy pipeline drugs are segmented as:
- In December 2016, NS Pharma, Inc.in collaboration with Nippon Shinyaku Co., Ltd., Cooperative International Neuromuscular Research Group (CINRG) and Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)commenced phase II multiple center, 2-period, randomized, placebo-controlled, dose finding studies of NS-O65/NCNP-O1 to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping
- In December 2014, Pfizer reported enrollment of first patient in a multicenter phase II clinical trial of its investigational compound PF-06252616 in boys with Duchenne Muscular Dystrophy. PF-06252616 is an experimental, infused, anti myostatin monoclonal antibody.PF-06252616 was granted Orphan Drug designation in July 2012 and fast track designation in November 2012 by U.S.FDA and in in 2013, PF-06252616 was granted Orphan medical product designation by European Medical Agency.
Muscular Dystrophy Disease Pipeline Drugs Assessment report studies the various therapeutics under clinical development for Muscular Dystrophy treatment along with targets for various drug candidate. The report provides plethora of information pertaining to trail phases, companies involved in the Muscular Dystrophy disease pipeline drugs development. This report studies the dynamics of the Muscular Dystrophy Disease 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 Muscular Dystrophy disease pipeline drugs assessment comprehensively presents the geographic location, trial status information along with key players involved in the therapeutics development.
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