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Determination of qPCR reference genes suitable for normalizing gene expression in a novel model of Duchenne muscular dystrophy, the D2-mdx mouse
published 13 Nov 2024
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Characterization of patients with Duchenne muscular dystrophy across previously developed health states
published 30 Oct 2024
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Distribution of MRI-derived T2 values as a biomarker for in vivo rapid screening of phenotype severity in mdx mice
published 19 Sep 2024
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Evaluation of dental manifestations in X-linked hypophosphatemia using orthopantomography
published 26 Jul 2024
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Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study
published 10 Jul 2024
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Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials
published 03 Jun 2024
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Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
published 16 May 2024
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Chloroquine decreases cardiac fibrosis and improves cardiac function in a mouse model of Duchenne muscular dystrophy
published 31 Jan 2024
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A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice
published 25 Jan 2024
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A missense mutation in the highly conserved TNF-like domain of Ectodysplasin A is the candidate causative variant for X-linked hypohidrotic ectodermal dysplasia in Limousin cattle: Clinical, histological, and molecular analyses
published 22 Jan 2024
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Adherence tool for prophylactic haemophilia treatment in adult and adolescent patients: A systematic review and meta-analysis protocol
published 14 Dec 2023
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The frequency of complications in a cohort of patients diagnosed with hemophilia A and hemophilia B receiving prophylactic treatment in Colombia: A retrospective noninterventional study
published 27 Nov 2023
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Identification of wheelchair seating criteria in adults with neuromuscular diseases: A Delphi study
published 08 Sep 2023
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