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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: 16 Nov 2023

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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

    • 27 Oct 2023 First clinical results from this trial published in the Atamyo Therapeutics Media Release
    • 27 Oct 2023 According to an Atamyo Therapeutics media release, company announced the Completion of enrollment of the first dose cohort of 3 patients
    • 27 Oct 2023 According to an Atamyo Therapeutics media release, company announced that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of this trial.With the DSMB clearance to start the 2nd cohort, company is looking forward to enroll new patients in Europe and in the US with a 3-times higher dose.
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