Recent Advancements Across Rare Inherited Neuromuscular Disorders

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Published: 16 Mar 2018

This edition of the Genetic Technology TOE depicts life sciences trends across rare inherited neuromuscular disorders. Innovative business and societal strategies involving comprehensive value assessment, stakeholders’ engagement, novel reimbursement approaches, and new manufacturing approaches for producing orphan drugs are critical to bypass many of the present technological, regulatory, and financial challenges. The corresponding clinical trials scenario for lysosomal storage disorders is also presented in the TOE. The Genetic Technology TechVision Opportunity Engine (TOE) presents the latest R&D developments and advances with an emphasis on opportunities to profit in the explosive field of genetic technology through technology transfer, joint ventures, and acquisitions. The complete gamut of genetic technology applications is covered including the latest developments in omics technologies; genetic, cellular, and alternative therapies; sequencing technologies; and genetically altered animals and plants. The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare. Keywords: Rare inherited neuromuscular disorders, lysosomal storage disorders, site-directed enzyme enhancement therapy, inborn errors of metabolism, x-linked adrenoleukodystrophy, MIN-102, peroxisome proliferator-activated receptor gamma agonist, cerebral adrenoleukodystrophy, adrenomyeloneuropathy, recombinant enzyme therapy, alpha-mannosidosis, aspartylglucosaminuria, leukodystrophy, globoid cell leukodystrophy, galactosyl-ceramide lipidosis, cyclin-dependent kinase-like 5, adeno associated virus gene therapy



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