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Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

The Journal of pediatrics, 2021-04, Vol.231, p.265-268

2021

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Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

Orphanet journal of rare diseases, 2017-11, Vol.12 (1), p.175-175, Article 175

2017

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SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy

PloS one, 2010-08, Vol.5 (8), p.e12140

2010

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Natural history of denervation in SMA: Relation to age, SMN2 copy number, and function

Annals of neurology, 2005-05, Vol.57 (5), p.704-712

2005

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Phase II open label study of valproic acid in spinal muscular atrophy

PloS one, 2009-05, Vol.4 (5), p.e5268-e5268

2009

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Alternating hemiplegia of childhood: early characteristics and evolution of a neurodevelopmental syndrome

Pediatrics (Evanston), 2009-03, Vol.123 (3), p.e534-e541

2009

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Treatment preferences in spinal muscular atrophy: A swing weighting study for caregivers of patients with SMA types 1 and 2

PloS one, 2024-10, Vol.19 (10), p.e0309666

2024

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Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program

Pediatric neurology, 2022-07, Vol.132, p.27-32

2022

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Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Drug safety, 2021-10, Vol.44 (10), p.1109-1119

2021

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Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial

Neuromuscular disorders : NMD, 2023-08, Vol.33 (8), p.670-676

2023

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Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA

Contemporary clinical trials communications, 2018-09, Vol.11, p.113-119

2018

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Outcome measures in a cohort of ambulatory adults with spinal muscular atrophy

Muscle & nerve, 2020-02, Vol.61 (2), p.187-191

2020

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Perspectives on Clinical Trials in Spinal Muscular Atrophy

Journal of child neurology, 2007-08, Vol.22 (8), p.957-966

2007

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Sodium channel mutations and susceptibility to heart failure and atrial fibrillation

JAMA : the journal of the American Medical Association, 2005-01, Vol.293 (4), p.447

2005

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Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Drug safety, 2022-02, Vol.45 (2), p.191-192

2022

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SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

PloS one, 2011-07, Vol.6 (7), p.e21296

2011

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SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy

Muscle & nerve, 2014-02, Vol.49 (2), p.187-192

2014

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Compound muscle action potential and motor function in children with spinal muscular atrophy

Muscle & nerve, 2010-11, Vol.42 (5), p.703-708

2010

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Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort

Journal of neuromuscular diseases, 2020-01, Vol.7 (1), p.33-40

2020

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Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study (S25.001)

Neurology, 2019-04, Vol.92 (15_supplement)

2019

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Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

Journal of neuromuscular diseases, 2023-01, Vol.10 (4), p.531-540

2023

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Clinical trial of L‐Carnitine and valproic acid in spinal muscular atrophy type I

Muscle & nerve, 2018-02, Vol.57 (2), p.193-199

2018

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Pregnancy and delivery in women with spinal muscular atrophy

International journal of neuroscience, 2017-11, Vol.127 (11), p.953-957

2017

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Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy

Natural history of denervation in SMA: Relation to age, SMN2 copy number, and function

Phase II open label study of valproic acid in spinal muscular atrophy

Alternating hemiplegia of childhood: early characteristics and evolution of a neurodevelopmental syndrome

Treatment preferences in spinal muscular atrophy: A swing weighting study for caregivers of patients with SMA types 1 and 2

Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

Natural history of infantile‐onset spinal muscular atrophy

Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial

Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA

Outcome measures in a cohort of ambulatory adults with spinal muscular atrophy

Perspectives on Clinical Trials in Spinal Muscular Atrophy

Sodium channel mutations and susceptibility to heart failure and atrial fibrillation

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy

Compound muscle action potential and motor function in children with spinal muscular atrophy

Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort

Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study (S25.001)

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

Clinical trial of L‐Carnitine and valproic acid in spinal muscular atrophy type I

Pregnancy and delivery in women with spinal muscular atrophy

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