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Plos One|June 27, 2025
Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophyFrancesco Muntoni, James Signorovitch, Nathalie Goemans, et al.
Neuromuscular Disorders : NMD|April 9, 2022
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controlsFrancesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Plos One|July 10, 2024
Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center studyFrancesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Neurology|February 1, 2023
<i>DMD</i> Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical TrialsFrancesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Transplantation and Cellular Therapy|September 30, 2022
Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord BloodChenyu Lin, Gautam Sajeev, Patrick J Stiff, et al.
Circulation|October 10, 2018
Genotype and Lifetime Burden of Disease in Hypertrophic Cardiomyopathy: Insights from the Sarcomeric Human Cardiomyopathy Registry (SHaRe)Carolyn Y Ho, Sharlene M Day, Euan A Ashley, et al.
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