Publications by authors named "Jean Mah"

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Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year children.
Swati Mummidivarpu, Utkarsh J Dang, Michael Ziemba, Yetrib Hathout, Paula R Clemens, Jean K Mah

Steroids· January 2026

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Feasibility of a Home-Based Exergaming Intervention for Youth With Spinal Muscular Atrophy.
Ihsane Iraqi, Pamela Ng, Xing Chen, Niamh Cushen, Juergen Gottowik, Jean K Mah

Muscle Nerve· December 2025

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Reviewer Comment on Slayter et al. "An Exploratory 12-Month Observational Study of Adults with Spinal Muscular Atrophy: Learning from Our Tools".
Tiffany R Price, Jean K Mah

Can J Neurol Sci· October 2025

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Patterns of Adherence to Lung Volume Recruitment Therapy Amongst Individuals With Duchenne Muscular Dystrophy: A Secondary Analysis of the STEADFAST Randomized Controlled Trial.
Naomi Dussah, Douglas A McKim, Jean K Mah, Hugh J McMillan, Craig Campbell

Pediatr Pulmonol· October 2025

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Lung Volume Recruitment and Quality of Life in Duchenne Muscular Dystrophy: Secondary Analysis of the STEADFAST Randomized Controlled Trial.
Anne Tsampalieros, Doug McKim, Nick Barrowman, Vid Bijelic, Jean K Mah

Ann Am Thorac Soc· August 2025

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Longitudinal Outcomes Among Patients With Duchenne Muscular Dystrophy: A Canadian Retrospective Population-Based Study.
Christina Qian, Alexa C Klimchak, Shelagh M Szabo, Katherine L Gooch, Roxana Dragan, Jean K Mah

Muscle Nerve· June 2025

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Therapeutic options for Duchenne muscular dystrophy: hope or hype?
Jean K Mah

Ther Adv Neurol Disord· June 2025

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Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen.
Guillaume Gauvreau, Tarannum Behlim, Pamela Ng, Victoria Hodgkinson, Kathryn Selby, Jean K Mah

Can J Neurol Sci· May 2025

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Equitable Access to Disease-Modifying Therapies for Canadian Children with SMA and Four Copies.
Hugh J McMillan, Hernan Gonorazky, Craig Campbell, Nicolas Chrestian, Megan Crone, Jean K Mah

Can J Neurol Sci· November 2024

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Macrostructural Brain Abnormalities in Spinal Muscular Atrophy: A Case-Control Study.
Emilie Groulx-Boivin, Andrea Oliveira-Carneiro, Helen Carlson, Amalia Floer, Adam Kirton, Jean Mah

Neurol Genet· October 2024

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A Study on the Incidence and Prevalence of 5q Spinal Muscular Atrophy in Canada Using Multiple Data Sources.
Tiffany R Price, Victoria Hodgkinson, Grace Westbury, Lawrence Korngut, Micheil A Innes, Jean K Mah

Can J Neurol Sci· September 2024

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Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Eugenio Mercuri, Juan J Vilchez, Odile Boespflug-Tanguy, Craig M Zaidman, Jean K Mah

Lancet Neurol· April 2024

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Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Utkarsh J Dang, Jesse M Damsker, Michela Guglieri, Paula R Clemens, Seth J Perlman, Jean K Mah

Neurology· March 2024

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Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Victor D Armengol, Basil T Darras, Ahmad A Abulaban, Ali Alshehri, Nina Barisic, Jean Mah

Neurol Clin Pract· February 2024

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Findings from the Longitudinal CINRG Becker Natural History Study.
Paula R Clemens, Heather Gordish-Dressman, Gabriela Niizawa, Ksenija Gorni, Michela Guglieri, Jean Mah

J Neuromuscul Dis· January 2024

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Alberta Spinal Muscular Atrophy Newborn Screening-Results from Year 1 Pilot Project.
Farshad Niri, Jessie Nicholls, Kelly Baptista Wyatt, Christine Walker, Tiffany Price, Jean K Mah

Int J Neonatal Screen· July 2023

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Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
Paula R Clemens, Vamshi K Rao, Anne M Connolly, Amy D Harper, Jean K Mah

J Neuromuscul Dis· May 2023

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Understanding the experiences of lung volume recruitment among boys with Duchenne muscular dystrophy: A multicenter qualitative study.
Sherri L Katz, Henrietta Blinder, Dennis Newhook, Leana Azerrad Bmus, Stuart Nicholls, Jean K Mah

Pediatr Pulmonol· January 2023

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Machine learning classification of multiple sclerosis in children using optical coherence tomography.
Beyza Ciftci Kavaklioglu, Lauren Erdman, Anna Goldenberg, Can Kavaklioglu, Cara Alexander, Jean K Mah

Mult Scler· December 2022

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Corneal nerve and nerve conduction abnormalities in children with type 1 diabetes.
Danièle Pacaud, Kenneth G Romanchuk, Heidi Virtanen, Maryam Ferdousi, Alberto Nettel-Aguirre, Jean K Mah

Pediatr Diabetes· December 2022

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Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy.
Sarah P Sherlock, Jeffrey Palmer, Kathryn R Wagner, Hoda Z Abdel-Hamid, Cuixia Tian, Jean K Mah

Sci Rep· November 2022

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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Michela Guglieri, Paula R Clemens, Seth J Perlman, Edward C Smith, Iain Horrocks, Jean K Mah

JAMA Neurol· October 2022

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Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.
Sarah P Sherlock, Jeffrey Palmer, Kathryn R Wagner, Hoda Z Abdel-Hamid, Enrico Bertini, Jean K Mah

J Neurol· August 2022

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Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial.
Francesco Muntoni, Michela Guglieri, Jean K Mah, Kathryn R Wagner, John F Brandsema

PLoS One· August 2022

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Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Paula R Clemens, Vamshi K Rao, Anne M Connolly, Amy D Harper, Jean K Mah

J Neuromuscul Dis· July 2022

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