Elizabeth A. Wraige

Guy's And St Thomas' NHS Foundation Trust
London, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 37 publications

Risk-benefit profile of onasemnogene abeparvovec in older and heavier children with spinal muscular atrophy type 1.

Risk-benefit profile of onasemnogene abeparvovec in older and heavier children with spinal muscular atrophy type 1.

Journal: Neuromuscular disorders : NMD

Published: June 07, 2024

Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment with onasemnogene abeparvovec - The SMA REACH UK network experience.

Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment with onasemnogene abeparvovec - The SMA REACH UK network experience.

Journal: Neuromuscular disorders : NMD

Published: March 28, 2024

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.

Journal: Journal of neuromuscular diseases

Published: January 08, 2024

Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.

Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.

Journal: The Lancet regional health. Europe

Published: September 26, 2023

Gene therapy for spinal muscular atrophy.

Gene therapy for spinal muscular atrophy.

Journal: Archives of disease in childhood. Education and practice edition

Published: March 21, 2023

The emerging spectrum of fetal acetylcholine receptor antibody-related disorders (FARAD).

The emerging spectrum of fetal acetylcholine receptor antibody-related disorders (FARAD).

Journal: Brain : a journal of neurology

Published: February 10, 2023

Long-term Natural History of Pediatric Dominant and Recessive RYR1-Related Myopathy.

Long-term Natural History of Pediatric Dominant and Recessive RYR1-Related Myopathy.

Journal: Neurology

Published: November 29, 2022

New treatments in spinal muscular atrophy.

New treatments in spinal muscular atrophy.

Journal: Archives of disease in childhood

Published: February 24, 2022

Digenic inheritance involving a muscle-specific protein kinase and the giant titin protein causes a skeletal muscle myopathy.

Digenic inheritance involving a muscle-specific protein kinase and the giant titin protein causes a skeletal muscle myopathy.

Journal: Nature genetics

Published: March 29, 2021

Genetic neuropathies presenting with CIDP-like features in childhood.

Genetic neuropathies presenting with CIDP-like features in childhood.

Journal: Neuromuscular disorders : NMD

Published: June 01, 2020

Making sense of missense variants in TTN-related congenital myopathies.

Making sense of missense variants in TTN-related congenital myopathies.

Journal: Acta neuropathologica

Published: May 19, 2020

RCPCH Stroke in Childhood Guidelines: how well are these being followed?

RCPCH Stroke in Childhood Guidelines: how well are these being followed?

Journal: Archives of disease in childhood

Published: April 27, 2020

Showing 1-12 of 37

Last Updated: 09/26/2026