Genetic linkage

Height development and multiple bone health indicators in children aged 2–12 years with Duchenne muscular dystrophy (DMD)

Bingying Wang, Linyuhan Zhou, [ ... ], Xiaomei Sun

Phenome-wide association network demonstrates close connection with individual disease trajectories from the HUNT study

Martina Hall, Marit K. Skinderhaug, Eivind Almaas

Determination of qPCR reference genes suitable for normalizing gene expression in a novel model of Duchenne muscular dystrophy, the D2-mdx mouse

Brigida Boccanegra, Roberta Lenti, [ ... ], Annamaria De Luca

Characterization of patients with Duchenne muscular dystrophy across previously developed health states

Francesco Muntoni, Nathalie Goemans, [ ... ], the PRO-DMD-01 study, the Imaging DMD study, iMDEX, the collaborative Trajectory Analysis Project (cTAP)

Distribution of MRI-derived T2 values as a biomarker for in vivo rapid screening of phenotype severity in mdx mice

Emily A. Waters, Chad R. Haney, [ ... ], Alexis R. Demonbreun

Evaluation of dental manifestations in X-linked hypophosphatemia using orthopantomography

Rena Okawa, Misato Takagi, [ ... ], Kazuhiko Nakano

Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study

Francesco Muntoni, James Signorovitch, [ ... ], cTAP

Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials

Craig M. McDonald, James Signorovitch, [ ... ], Investigators from the PRO-DMD-01 Study, Collaborative Trajectory Analysis Project (cTAP)

Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

Linda P. Lowes, Lindsay N. Alfano, [ ... ], Jerry R. Mendell

Chloroquine decreases cardiac fibrosis and improves cardiac function in a mouse model of Duchenne muscular dystrophy

Takuya Hirata, Shiro Baba, [ ... ], Junko Takita

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

Ian R. Woodcock, George Tachas, [ ... ], Eppie M. Yiu

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

Frederik Krull, Martina Bleyer, Jana Schäfer, Bertram Brenig

Adherence tool for prophylactic haemophilia treatment in adult and adolescent patients: A systematic review and meta-analysis protocol

Fadzlin Mohd Mokhtar, Sutha Rajakumar, Hasniza Zaman Huri

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