Suchergebnisse - "Shieh, Perry B"

PubPharm (7)

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

4

Incorporating Spinal Muscular Atrophy Analysis by Next-Generation Sequencing into a Comprehensive Multigene Panel for Neuromuscular Disorders

enthalten in: Genetic testing and molecular biomarkers | 2020

von Tan, C. | Westbrook, M. | Truty, R. | +4

5

Revised Recommendations for the Treatment of Infants Diagnosed with Spinal Muscular Atrophy Via Newborn Screening Who Have 4 Copies of SMN2

enthalten in: Journal of neuromuscular diseases | 2020

von Glascock, J. | Sampson, J. | Connolly, A. | +11

6

Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening

enthalten in: Journal of neuromuscular diseases | 2018

von Glascock, J. | Sampson, J. | Haidet-Phillips, A. | +12

CommentIn: Pediatr Neurol Briefs. 2019 Dec 31;33:5. - PMID 31929716

7

Natural history of infantile-onset spinal muscular atrophy

enthalten in: Annals of neurology | 2017

von Kolb, S. | Coffey, C. | Yankey, J. | +35

CommentIn: Ann Neurol. 2017 Dec;82(6):857-858. - PMID 29149769

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