RAAV9-hFKRPco_miR-208a ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
113筋ジストロフィー1

113. 筋ジストロフィー


臨床試験数 : 646 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
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agemin
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PhaseCountries
1EUCTR2021-004276-33-DK
(EUCTR)
11/02/202201/10/2021GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophyA 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) FKRP-related limb-girdle muscular dystrophy (LGMD R9)
MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: rAAV9-hFKRPco_miR-208a
Product Code: GNT0006
INN or Proposed INN: not yet available
Other descriptive name: Adeno-associated virus serotype 9 carrying the human fukutin-related protein and target sequence of the miR-208a
Atamyo TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 1;Phase 2France;United States;Canada;Denmark;Norway;Germany;United Kingdom;Sweden