Recent Advancements Across Rare Inherited Neuromuscular Disorders

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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 technolo

Table of Contents

Recent Advancements Across Rare Inherited Neuromuscular DisordersEnhancing Therapeutics Associated with Rare Inherited Neuromuscular Disorders Site-directed Enzyme Enhancement Therapy for Inherited DiseasesRecombinant Enzyme Therapy for Lysosomal Storage DisordersComputational Biology and Machine Learning for Orphan DiseasesAdeno Associated Virus Gene Therapy for Neuromuscular Diseases Clinical Trials and Industry InteractionsInterventional Studies Industry Interactions




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