Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
This opportunity funds translational, IND-enabling work to prepare first-in-human testing of gene-based or transcript-directed therapies for ultra-rare neurological and neuromuscular disorders. Supported activities include proving biological rationale and preparing a clinical candidate with strong proof of concept in a model system relevant to the target patient population, with the aim of ena…
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