Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy

Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

(2019) Omar Dabbous et al.   ADVANCES IN THERAPY

Impact of age and motor function in a phase 1/2A study of infants with SMA Type 1 receiving single-dose gene replacement therapy

(2019) Linda P. Lowes et al.   PEDIATRIC NEUROLOGY

Onasemnogene Abeparvovec: First Global Approval

(2019) Sheridan M. Hoy   DRUGS

From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1

(2019) Samiah A. Al-Zaidy et al.   PEDIATRIC NEUROLOGY

Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164–76. doi:10.1007/s12325-019-00923-8

(2019) Alfred W. Sandrock et al.   ADVANCES IN THERAPY

Combination therapy with nusinersen and AVXS-101 in SMA type 1

(2019) Bo Hoon Lee et al.   NEUROLOGY

Nusinersen: A Treatment for Spinal Muscular Atrophy

(2018) Melanie K. Claborn et al.   ANNALS OF PHARMACOTHERAPY

Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care

(2018) Eugenio Mercuri et al.   NEUROMUSCULAR DISORDERS

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

(2018) Samiah Al‐Zaidy et al.   PEDIATRIC PULMONOLOGY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

(2016) Stephen J. Kolb et al.   Annals of Clinical and Translational Neurology

Spinal muscular atrophy

(2011) Adele D'Amico et al.   Orphanet Journal of Rare Diseases

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

(2010) Aga Lewelt et al.   MUSCLE & NERVE

The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): Test development and reliability

(2010) A.M. Glanzman et al.   NEUROMUSCULAR DISORDERS

Spinal muscular atrophy

(2008) Mitchell R Lunn et al.   LANCET