Publications by authors named "E Wraige"

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Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment with onasemnogene abeparvovec - The SMA REACH UK network experience.
Amy Wolfe, Jennie Sheehan, Alex Schofield, Helen Cranney, Emer O'Reilly, Elizabeth Wraige

Neuromuscul Disord· November 2024

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Risk-benefit profile of onasemnogene abeparvovec in older and heavier children with spinal muscular atrophy type 1.
Rebecca Finnegan, Adnan Manzur, Pinki Munot, Anil Dhawan, Archana Murugan, Elizabeth Wraige

Neuromuscul Disord· September 2024

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Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.
Nikki Cornell, Anne-Marie Childs, Elizabeth Wraige, Pinki Munot, Gautam Ambegaonkar

J Neuromuscul Dis· March 2024

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Digenic inheritance involving a muscle-specific protein kinase and the giant titin protein causes a skeletal muscle myopathy.
Ana Töpf, Dan Cox, Irina T Zaharieva, Valeria Di Leo, Jaakko Sarparanta, Elizabeth Wraige

Nat Genet· March 2024

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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.
Vasantha Gowda, Mark Atherton, Archana Murugan, Laurent Servais, Jennie Sheehan, Elizabeth Wraige

Lancet Reg Health Eur· February 2024

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