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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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Adherence to hemophilia patients with prophylaxis: Veritas-Pro and psychometric properties adapted to Turkish
published 09 Aug 2023
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Quality of life, self-reported outcomes and impact of education among people with moderate and severe hemophilia A: An integrated perspective from a Latin American country
published 06 Jul 2023
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Duchenne muscular dystrophy in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact
published 27 Jun 2023
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Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy
published 26 Apr 2023
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Rett syndrome severity estimation with the BioStamp nPoint using interactions between heart rate variability and body movement
published 01 Mar 2023
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Antisense oligonucleotides to therapeutically target SARS-CoV-2 infection
published 03 Feb 2023
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