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MYO 201

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Drug Profile

MYO 201

Alternative Names: AAVrh-74-MHCK7-DYSF; AAVrh.74.MHCK7.DYSF; MYO201; SRP-6004

Latest Information Update: 22 Jul 2025

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At a glance

  • Originator Nationwide Children's Hospital
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I Limb girdle muscular dystrophy type 2B

Most Recent Events

  • 18 Jul 2025 US FDA places clinical hold on all clinical trials for limb girdle muscular dystrophy using AAVrh74 gene therapies
  • 27 Mar 2024 Pharmacodynamics data from a preclinical trial in Limb-girdle muscular dystrophy type 2B presented at the 125th Annual Meeting of the American Society for Clinical Pharmacology and Therapeutics (ASCPT-2024)
  • 22 May 2023 Phase-I clinical trials in Limb girdle muscular dystrophies type 2B (LGMD2B) in USA (IV) (NCT05906251)

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