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Rett syndrome

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Rare diseases
FDA Signs Off on Neurogene’s ‘Best-Case Scenario’ Pivotal Study Design for Rett Syndrome
The pivotal trial for Neurogene’s Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that could help ensure a broader label for the drug product, if approved, according to analysts.
July 1, 2025
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2 min read
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Tristan Manalac
PRESS RELEASES
Press Releases
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
March 6, 2025
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8 min read
Press Releases
Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
November 18, 2024
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7 min read
Press Releases
Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
November 12, 2024
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11 min read
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