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  • Therapeutics for Rare Neuromuscular Diseases: Which Growth Opportunities Showcase Vast Potential?
    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 present significant clinical challenges because of disease heterogeneity and rarity of occurrence. The lack of measurable early disease markers and clinical outcomes further complicates the management of such disorders.

    • Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class.

    • An overview of the global clinical trial landscape for emerging Phase 1, 2, and 3 treatments revealed the key hotspots for clinical analyses.

    • These indications are likely to witness new drug developments. It explores emerging biologics across these six NMDs.

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