Dystrophin Market:
Research Insights:
Rising prevalence of musculoskeletal and genetic disorders stimulating the demand for DMD drugs
Dystrophin Market is a group of proteins found in muscle cells that function together to fortify muscle fibers and protect them from injuries and strain-related damages caused during muscle contraction. It is a rod-shaped cytoplasmic protein that is an essential component of the protein complex called costamere, which connects the cytoskeleton of a muscle fiber with the surrounding extracellular matrix. Dystrophin is located between the sarcolemma and myofilaments present in the muscle fiber, called myofiber. The deficiency of dystrophin decreases muscle fiber strength, reduces muscle stiffness, and enhances the risks of sarcolemmal deformability. Moreover, dystrophin deficiency has been associated with conditions such as Duchenne and Becker muscular dystrophies that lead to progressive muscular weakness. Frame-shift mutations resulting from the deletion of exons from the dystrophin gene lead to Duchenne muscular dystrophy (DMD).
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The global dystrophin market has gained enormous traction in the past years, owing to a plethora of market growth-inducing factors. These factors include the growing incidence of musculoskeletal and genetic disorders, rising prevalence of Duchenne muscular dystrophy, the burgeoning demand for DMD drugs worldwide, the surging adoption of mutation-targeted therapies, favorable government initiatives towards dystrophin drug development, and increasing investments in research & development. The most commonly prescribed dystrophin drugs include Biostrophin, Exondys51, Emflaza, Translarna, DS-5141, NS-065, BMBD-001, and numerous others. Several key market players, including some healthcare giants, have introduced various approaches to drug development, such as anti-inflammatory therapy, exon skipping, stop-codon read-through, and numerous others. Therefore, the emergence of these effective mutation-specific therapies has created several growth prospects for the global dystrophin market over the upcoming years. However, stringent regulatory norms regarding drug development procedures and the lack of standardized protocols for clinical efficacy determination are some of the key factors posing threats to the global market expansion.
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Among several key regions, North America is the leading market for dystrophin due to the high prevalence of muscular dystrophy, rising government funding for R&D activities in DMD, favorable medical reimbursement policies, soaring healthcare expenditure, and the launch of promising pipeline drug candidates. According to market analysts, the U.S. is the leading growth engine for the North American dystrophin market. Pfizer Inc., Bristol-Myers Squibb, Solid Biosciences Inc., Wave Life Sciences, Asklepios BioPharmaceutical, Inc., Catabasis Pharmaceuticals, Sarepta Therapeutics, BioMarin Pharmaceutical Inc., Editas Medicine Inc., and PTC Therapeutics are the key rivals in the global dystrophin market. In October 2020, pharmaceutical giant Pfizer announced that its investigational gene therapy candidate for DMD, named PF-06939926, received the Fast Track Designation from the U.S. FDA on the basis of the positive Phase1b trial of the drug. In July 2020, the leading U.S.-based biopharmaceutical company, Sarepta Therapeutics, received the Fast Track Designation by the FDA for its micro-dystrophin gene therapy candidate Srp-9001 following the drug’s successful placebo-controlled study. Furthermore, in August 2020, the U.S. FDA approved the use of N.S. Pharma’s VILTEPSO (Viltolarsen) injection for treating DMD in patients with a confirmed mutation of the DMD gene amenable to exon 53 skipping.
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