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As cell and gene therapy (CGT) moves from ultra-rare indications to more common indications, manufacturing capabilities will have to expand to support the commercial requirements of these diseases. Contract research organizations (CROs) can play a critical role in long-term genetic analysis and monitor safety and efficacy up to 15 years after treat...

09 Nov 2022  |  Global  |  Technology Research

Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis

Strategic partnering and new product development will enable better biologics for personalized and targeted treatment

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Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs). The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies. NMDs prese...