New Gene Therapy Changes Treatment Landscape for Metachromatic Leukodystrophy

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Nelson, Roxanne
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10.1002/ajmg.a.63276
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Q3 [遗传学];
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071007 ; 090102 ;
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页数:2
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  • [1] [Anonymous], 2024, Press release
  • [2] A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel
    Armstrong, Nigel
    Olaye, Andrew
    Noake, Caro
    Pang, Francis
    [J]. ORPHANET JOURNAL OF RARE DISEASES, 2023, 18 (01)
  • [3] Orchard PJ., Compassionate use of lentiviral gene therapy for metachromatic leukodystrophy. WORLDSymposium presentation, February 7