Kristy J. Rose

Central Parkway
Newcastle Upon Tyne, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 27 publications

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

Journal: The Lancet. Child & adolescent health

Published: January 28, 2026

Performance of upper limb entry item to predict forced vital capacity in dysferlin-deficient limb girdle muscular dystrophy.

Performance of upper limb entry item to predict forced vital capacity in dysferlin-deficient limb girdle muscular dystrophy.

Journal: Neuromuscular disorders : NMD

Published: March 26, 2024

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.

Journal: The Lancet. Neurology

Published: April 08, 2022

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

Journal: Frontiers in neurology

Published: December 03, 2021

Clinical practice guideline for the management of paediatric Charcot-Marie-Tooth disease.

Clinical practice guideline for the management of paediatric Charcot-Marie-Tooth disease.

Journal: Journal of neurology, neurosurgery, and psychiatry

Published: November 18, 2021

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.

Journal: The New England journal of medicine

Published: July 28, 2021

Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases.

Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases.

Journal: Frontiers in genetics

Published: July 04, 2021

Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic.

Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic.

Journal: Frontiers in genetics

Published: July 02, 2021

Risdiplam in Type 1 Spinal Muscular Atrophy.

Risdiplam in Type 1 Spinal Muscular Atrophy.

Journal: The New England journal of medicine

Published: February 24, 2021

Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.

Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.

Journal: Annals of neurology

Published: April 24, 2020

Erratum to: Development and validation of the Charcot-Marie-Tooth Disease Infant Scale.

Erratum to: Development and validation of the Charcot-Marie-Tooth Disease Infant Scale.

Journal: Brain : a journal of neurology

Published: January 17, 2019

Physical activity of children and adolescents with Charcot-Marie-Tooth neuropathies: A cross-sectional case-controlled study.

Physical activity of children and adolescents with Charcot-Marie-Tooth neuropathies: A cross-sectional case-controlled study.

Journal: PloS one

Published: December 06, 2018

Showing 1-12 of 27

Last Updated: 09/26/2026