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A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I)

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A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I)

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 02 Oct 2024

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At a glance

  • Drugs ATA 100 (Primary)
  • Indications Limb girdle muscular dystrophies
  • Focus First in man; Therapeutic Use
  • Sponsors Atamyo Therapeutics

    • Most Recent Events

    • 30 Sep 2024 According to an Atamyo Therapeutics media release, the company announces the enrollment of the last patient in the dose-escalation phase of its Phase 1b clinical trial of ATA-100, Updated results of the clinical trial will be featured in an oral presentation at the 29th International Annual Congress of the World Muscle Society (WMS)
    • 16 Sep 2024 According to an Atamyo Therapeutics media release, two communications on Atamyo's LGMD programs will be presented at the ASGCT's Breakthroughs in Muscular Dystrophy Conference, November 19-20, 2024, in Chicago, IL, in oral presentation entitled "Development of Gene Therapy for LGMD-R9." on 19 Nov 2024 and a poster "Overcoming Phenotypic Variability in Dystrophic Models: A Machine Learning Method for Refined Gene Therapy Evaluation." on 16 Nov 2024.
    • 16 Sep 2024 According to an Atamyo Therapeutics media release, S Olivier (Atamyo) et al., will present a poster (Poster number P0264) entitled "Natural history of limb girdle muscular dystrophy R9: two-year follow-up of a European cohort." at the European Society of Gene & Cell Therapy 31st Annual Congress, October 22-25, 2024 in La Nuvola, Rome, Italy.

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