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"Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)" is currently closed and not accepting applications.
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is sponsored by National Institute of Neurological Disorders and Stroke (NINDS) - NIH.
This program supports the development of state-of-the-art gene-based therapies for ultra-rare neurological and neuromuscular diseases from late-stage pre-clinical development into first-in-human clinical testing. Citrullinemia, as a rare metabolic disorder with neurological manifestations, could be relevant.
The program aims to accelerate discoveries into the clinic, provide resources and expertise, deliver therapeutics to patients, and standardize best practices. It supports PIs with a lead gene therapy candidate from start-to-finish over a 3-year period.
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Or search similar grants →According to the current listing, eligibility includes: Not explicitly stated beyond Principal Investigators (PIs) with a lead gene therapy candidate. Focus is on ultra-rare neurological and neuromuscular disorders affecting as few as one in fifty thousand people. Confirm the full requirements in the official notice before applying.
The published deadline was April 27, 2026, which has passed. Check the official notice for any future application windows before investing time in a proposal.
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is funded by National Institute of Neurological Disorders and Stroke (NINDS) - NIH. Verify program details on the funder's official page before applying.
Start from the official opportunity page linked in this listing — it carries the sponsor's submission instructions.
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