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ASPIRE Study: A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females Aged >2 to <4 Years With Rett Syndrome

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Trial Profile

ASPIRE Study: A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females Aged >2 to <4 Years With Rett Syndrome

Status: Not yet recruiting
Phase of Trial: Phase III

Latest Information Update: 23 Mar 2026

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At a glance

  • Drugs TSHA 102 (Primary)
  • Indications Rett syndrome
  • Focus Adverse reactions; Registrational
  • Acronyms ASPIRE
  • Sponsors Taysha Gene Therapies

Most Recent Events

  • 23 Mar 2026 New trial record

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