Muscular Dystrophy Disease Pipeline Drugs Assessment: By Trial Phase ( Preclinical Trials, Phase 1, Phase 2, Phase 3, Phase 4), By Drugs (Blinatumomab, Carfilzomib, calaspargasepegol, Dasatinib, Ibrutinib, Inotuzumabozogamicin, Ofatumumab, Ponatinib, Others), By Route of Administration (Oral, Parenteral)  

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

 Key Developments:

• 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.