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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
Taysha Gene Therapies Announces Details for Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting Reviewing Recent Updates from the TSHA-102 Clinical Program
June 4, 2025
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7 min read
Press Releases
Taysha Gene Therapies Announces Pivotal Part B Trial Design Details for TSHA-102 in Rett Syndrome Enabled by IRSF Natural History Data and Positive Clinical Data from Part A of the REVEAL Adult/Adolescent and Pediatric Trials Evaluating TSHA-102
May 29, 2025
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12 min read
Press Releases
Neurogene Announces Evidence-Based Monitoring and Treatment Intended to Reverse Rare Hyperinflammatory Syndrome Associated with High-Dose AAV
May 16, 2025
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5 min read
Press Releases
Neurogene Reports First Quarter 2025 Financial Results and Highlights Recent Updates
May 12, 2025
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9 min read
Press Releases
Apertura Gene Therapy and Rett Syndrome Research Trust Collaborate to Pioneer Advanced Genetic Medicines for Rett Syndrome Using TfR1-Targeted AAV Capsid
April 30, 2025
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4 min read
Press Releases
Neurogene Announces Peer-Reviewed Publication Demonstrating Gene Therapy Candidate NGN-401 with EXACT™ Technology Regulates Transgene Expression in Preclinical Models of Rett Syndrome
April 3, 2025
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8 min read
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