Suchergebnisse - "Shieh, Perry B"

PubPharm (3)

1

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1 : the Phase III SPR1NT trial

enthalten in: Nature medicine | 2022

von Strauss, K. | Farrar, M. | Muntoni, F. | +17

CommentIn: Nat Med. 2022 Jul;28(7):1348-1349. - PMID 35840728

2

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy : the Phase III SPR1NT trial

enthalten in: Nature medicine | 2022

von Strauss, K. | Farrar, M. | Muntoni, F. | +18

CommentIn: Nat Med. 2022 Jul;28(7):1348-1349. - PMID 35840728

3

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE) : an open-label, single-arm, multicentre, phase 3 trial

enthalten in: The Lancet. Neurology | 2021

von Day, J. | Finkel, R. | Chiriboga, C. | +18

CommentIn: Lancet Neurol. 2021 Apr;20(4):251-252. - PMID 33743228

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