Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry

Autor: Joanne M. Donovan, Ann Martin, Ann Lucas, Maria Cecilia Mancini, Leslie Cowen

Publikováno v: BMC Neurology
BMC Neurology, Vol 19, Iss 1, Pp 1-10 (2019)

Background Treatment options for Duchenne muscular dystrophy remain limited, although consensus treatment guidelines recommend corticosteroid use. Methods This retrospective analysis assessed corticosteroid use in ambulatory and nonambulatory US male

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a56b467111599e3f48a7e757ab7bbf60
http://europepmc.org/articles/PMC6498563

A Patient-Report Registry to Study Outcomes in a Rare Genetic Disease: DuchenneConnect

Autor: Silverstein, Cheri Alissa

Publikováno v: Silverstein, Cheri Alissa. (2013). A Patient-Report Registry to Study Outcomes in a Rare Genetic Disease: DuchenneConnect. UCLA: Clinical Research 0853. Retrieved from: http://www.escholarship.org/uc/item/0bc7q41m

Background: Duchenne Muscular Dystrophy (DMD) is the most common muscular dystrophy, but it is a rare disease, creating challenges for study design. Randomized trials have shown that steroids improve surrogate endpoints of skeletal muscle function, b

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od_______325::c5b71574e4bf221498c6df04d4987f95
http://n2t.net/ark:/13030/m50c50kp