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X-linked traits

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

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

Jorge E. Machado Alba, Juan David Wilches-Gutierrez, [ ... ], Manuel E. Machado-Duque

Identification of wheelchair seating criteria in adults with neuromuscular diseases: A Delphi study

Elise Dupitier, Manon Voisin, [ ... ], Samuel Pouplin

Adherence to hemophilia patients with prophylaxis: Veritas-Pro and psychometric properties adapted to Turkish

Zuhal Mehrekula Demirci, Nilufer Demiral Yilmaz, [ ... ], Fahri Sahin

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

Liliana Torres, Oscar Peñuela, [ ... ], Juan-Sebastian Franco

Duchenne muscular dystrophy in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact

Massimiliano Orso, Antonio Migliore, [ ... ], Marika Pane

Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy

Vandana Ayyar Gupta, Jacqueline M. Pitchforth, [ ... ], on behalf of the iMDEX Consortium and the U.K. NorthStar Clinical Network

Rett syndrome severity estimation with the BioStamp nPoint using interactions between heart rate variability and body movement

Pradyumna Byappanahalli Suresha, Heather O’Leary, [ ... ], Gari D. Clifford

Antisense oligonucleotides to therapeutically target SARS-CoV-2 infection

Yuanyuan Qiao, Jesse W. Wotring, [ ... ], Arul M. Chinnaiyan

Adherence to prophylaxis and bleeding outcome: A multicenter Nigerian study

Theresa Ukamaka Nwagha, Helen Chioma Okoye, [ ... ], Nnamdi Joel Osuji

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