Works about DUCHENNE muscular dystrophy


Results: 3066
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    Genetics.

    Published in:
    Indian Pediatrics, 2025, v. 62, n. 7, p. 549, doi. 10.1007/s13312-025-00092-8
    By:
    • Shriyan, Rhea
    Publication type:
    Article
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    Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy.

    Published in:
    PLoS ONE, 2025, v. 20, n. 6, p. 1, doi. 10.1371/journal.pone.0325736
    By:
    • Muntoni, Francesco;
    • Signorovitch, James;
    • Goemans, Nathalie;
    • Manzur, Adnan Y.;
    • Done, Nicolae;
    • Sajeev, Gautam;
    • Li, Jiayang;
    • Akbarnejad, Hanane;
    • Sharma, Aarushi;
    • Ward, Susan J.;
    • Niks, Erik H.;
    • Servais, Laurant;
    • Mercuri, Eugenio;
    • Guglieri, Michela;
    • Straub, Volker;
    • de Groot, Imelda;
    • Ridout, Deborah;
    • McDonald, Craig
    Publication type:
    Article
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    Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.

    Published in:
    2020
    By:
    • Smith, Edward C.;
    • Conklin, Laurie S.;
    • Hoffman, Eric P.;
    • Clemens, Paula R.;
    • Mah, Jean K.;
    • Finkel, Richard S.;
    • Guglieri, Michela;
    • Tulinius, Mar;
    • Nevo, Yoram;
    • Ryan, Monique M.;
    • Webster, Richard;
    • Castro, Diana;
    • Kuntz, Nancy L.;
    • Kerchner, Laurie;
    • Morgenroth, Lauren P.;
    • Arrieta, Adrienne;
    • Shimony, Maya;
    • Jaros, Mark;
    • Shale, Phil;
    • Gordish-Dressman, Heather
    Publication type:
    journal article
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    Gene Therapy in Pediatric Orthopedics.

    Published in:
    Osteology, 2024, v. 4, n. 1, p. 33, doi. 10.3390/osteology4010003
    By:
    • Olaonipekun, Emmanuel;
    • Lisyansky, Anthony;
    • Olaonipekun, Robin;
    • Merabia, Bouchra Ghania;
    • Gaber, Karim;
    • Kishta, Waleed
    Publication type:
    Article
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