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X-linked traits

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

Dp71 and intellectual disability in Indonesian patients with Duchenne muscular dystrophy

Kristy Iskandar, Agung Triono, [ ... ], Gunadi

Cognitive and behavioral functioning in two neurogenetic disorders; how different are these aspects in Duchenne muscular dystrophy and Neurofibromatosis type 1?

Danique M. J. Hellebrekers, Sandra A. M. van Abeelen, [ ... ], Johan S. H. Vles

Multiple criteria decision analysis for therapeutic innovations in a hemophilia care center: A pilot study of the organizational impact of innovation in hemophilia care management

Karen Beny, Amélie Dubromel, [ ... ], Claude Dussart

Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial

Francesco Muntoni, Michela Guglieri, [ ... ], Eugenio Mercuri

Age, corticosteroid treatment and site of mutations affect motor functional changes in young boys with Duchenne Muscular Dystrophy

Giorgia Coratti, Jacopo Lenkowicz, [ ... ], for the italian DMD study group

Optimization and evaluation of a two-stage chromogenic assay procedure for measurement of emicizumab plasma levels

Nasim Shahidi Hamedani, Johannes Oldenburg, Bernd Pötzsch, Jens Müller

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