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Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy.
Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM; Eteplirsen Investigators and the CINRG DNHS Investigators. Khan N, et al. Among authors: mcdonald cm. J Neuromuscul Dis. 2019;6(2):213-225. doi: 10.3233/JND-180351. J Neuromuscul Dis. 2019. PMID: 30856119 Free PMC article.
The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures.
Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM; CINRG Investigators. Henricson EK, et al. Among authors: mcdonald cm. Muscle Nerve. 2013 Jul;48(1):55-67. doi: 10.1002/mus.23808. Epub 2013 May 6. Muscle Nerve. 2013. PMID: 23649481 Free PMC article.
304 results