Results 241 to 250 of about 71,442 (257)

Fighting for every beat: cardiac therapies in Duchenne muscular dystrophy. [PDF]

Skelet Muscle
Muchir A.
europepmc   +1 more source

Histone deacetylases in Duchenne muscular dystrophy: a role in the mechanism of disease and a target for inhibition. [PDF]

Clin Epigenetics
Bertoldi M, Albamonte E, Bello L, D'amico A, Masson R, Nigro V, Pane M, Panicucci C, Sframeli M, Ricci F.   +9 more
europepmc   +1 more source

The Role of Systemic Inflammatory Indices in Predicting Cardiovascular Involvement in Children with Duchenne Muscular Dystrophy. [PDF]

Children (Basel)
Altınok Eİ, Kasar T.
europepmc   +1 more source

Endocrine and metabolic complications in a national cohort of Slovene children and adolescents with Duchenne muscular dystrophy: real-world criteria for transition to vamorolone therapy. [PDF]

Front Endocrinol (Lausanne)
Jazbinšek S, Vrščaj E, Šuput Omladič J, Butenko T, Loboda T, Kotnik P, Osredkar D.   +6 more
europepmc   +1 more source

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Assessing the Appropriateness of the EQ-5D for Duchenne Muscular Dystrophy: A Patient-Centered Study

Medical decision making, 2021
Background Duchenne muscular dystrophy is a rare degenerative neuromuscular disorder with pediatric onset. Recent approvals in Duchenne have placed attention on the economic evaluation in pricing and reimbursement decisions across a range of rare ...
Norah L. Crossnohere, R. Fischer, A. Lloyd, L. Prosser, J. Bridges   +4 more
semanticscholar   +1 more source

Neurodevelopmental, behavioral, and emotional symptoms common in Duchenne muscular dystrophy

Muscle and Nerve, 2020
We studied neurodevelopmental and behavioral/emotional symptoms in patients with Duchenne muscular dystrophy (DMD).
A. Darmahkasih, I. Rybalsky, C. Tian, K. Shellenbarger, P. Horn, J. Lambert, B. Wong   +6 more
semanticscholar   +1 more source

Golodirsen for Duchenne muscular dystrophy.

Drugs of Today, 2020
Duchenne muscular dystrophy (DMD) is a life-shortening X-linked genetic disorder characterized by progressive wasting and weakening of muscles in boys. Loss-of-function mutations in the DMD gene, which codes for dystrophin, lead to this disease.
S. Anwar, T. Yokota
semanticscholar   +1 more source