Article
The CINRG Becker Natural History Study: Baseline characteristics.
Muscle & nerve - 1 Sept 2020
Clemens Paula R, Niizawa Gabriela, Feng Jia, Florence Julaine, DʼAlessandro Andrea Smith, Morgenroth Lauren P, Gorni Ksenija, Guglieri Michela, Connolly Anne, Wicklund Matthew, Bertorini Tulio, Mah Jean K, Thangarajh Mathula, Smith Edward, Kuntz Nancy, McDonald Craig M, Henricson Erik K, Upadhyayula Saila, Byrne Barry, Manousakis Georgios, Harper Amy, Bravver Elena, Iannaccone Susan, Spurney Christopher, Cnaan Avital, Gordish-Dressman Heather
Abstract excerpt
We performed an observational, natural history study of males with in-frame dystrophin gene deletions causing Becker muscular dystrophy (BMD). A prospective natural history study collected longitudinal medical, strength, and timed function assessments. Eighty-three participants with genetically confirmed BMD were enrolled (age range 5.6-75.4 years). Lower extremity function and the percentage of participants who...
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