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Rett syndrome
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
Neurogene Reports First Quarter 2026 Financial Results and Highlights Recent Updates
May 13, 2026
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11 min read
Press Releases
Unravel Biosciences Announces First Patient Dosed in RVL-001 Proof of Concept Studies for Rett Syndrome and Pitt Hopkins Syndrome
May 12, 2026
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3 min read
Press Releases
Neurogene Highlights Data at ASGCT Meeting Supporting Therapeutic Rationale for ICV Delivery Method in CNS-targeted Gene Therapy
May 12, 2026
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8 min read
Press Releases
Neurogene Announces Upcoming Presentation on the Therapeutic Rationale for ICV Delivery in CNS-Targeted Gene Therapy
April 28, 2026
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7 min read
Press Releases
Taysha Gene Therapies to Present New Preclinical Data Supporting Construct Design of TSHA-102 for Rett Syndrome at the ASGCT 2026 Annual Meeting
April 28, 2026
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7 min read
Press Releases
Acadia Pharmaceuticals to Present Data at the 2026 American Academy of Neurology (AAN) Annual Meeting
April 19, 2026
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8 min read
Press Releases
Acadia Pharmaceuticals Announces DAYBUE® STIX (trofinetide) is Now Broadly Available in the United States for the Treatment of Rett Syndrome
April 7, 2026
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11 min read
Press Releases
Taysha Gene Therapies Reports Full-Year 2025 Financial Results and Provides Corporate Update
March 19, 2026
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13 min read
Press Releases
ProQR Announces Year End 2025 Operating and Financial Results
March 12, 2026
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10 min read
Press Releases
Acadia Pharmaceuticals Announces Plan to Request Re-Examination Following Negative CHMP Opinion for Trofinetide for the Treatment of Rett Syndrome
March 3, 2026
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7 min read
Press Releases
Neurogene Announces FDA Breakthrough Therapy Designation for NGN-401 Gene Therapy for Rett Syndrome
February 27, 2026
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7 min read
Press Releases
Evox Therapeutics Partners with Rett Syndrome Research Trust to Evaluate ExoEdit® Technology to Enable Gene Editing in Rett Syndrome
February 26, 2026
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3 min read
Press Releases
Acadia Pharmaceuticals Provides Update on Regulatory Submission for Trofinetide for the Treatment of Rett Syndrome in the European Union
February 3, 2026
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7 min read
Press Releases
Neurogene Announces Recent Achievements and Anticipated 2026 Key Milestones for NGN-401, a Potential Best-in-Class Gene Therapy for Rett Syndrome
January 12, 2026
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8 min read
Press Releases
ProQR Announces Encouraging AX-0810 Phase 1 Safety and PK Data, Development Candidate Selections, and 2026 Outlook
January 8, 2026
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11 min read
Press Releases
Taysha Gene Therapies Announces Progress Across TSHA-102 Pivotal Gene Therapy Program in Rett Syndrome
January 6, 2026
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7 min read
Press Releases
Acadia Pharmaceuticals Announces FDA Approval of DAYBUE® STIX (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of Rett Syndrome
December 15, 2025
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9 min read
Press Releases
Profluent Bio Announces Strategic Collaboration with Ensoma to Develop AI-Designed Base Editors for In Vivo Hematopoietic Stem Cell Therapies
December 10, 2025
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2 min read
Press Releases
Unravel Biosciences Announces Approval by Colombian Health Regulatory Authority to Initiate RVL-001 Clinical Studies for Rett Syndrome and Pitt Hopkins Syndrome
December 3, 2025
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2 min read
Press Releases
Profluent and RSRT Partner to Tackle Rett Syndrome with AI-Designed Custom Genomic Medicines
November 18, 2025
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3 min read
