Article
Cyclosporine A in Ullrich congenital muscular dystrophy: long-term results.
Oxidative medicine and cellular longevity - 1 Jan 2011
Merlini Luciano, Sabatelli Patrizia, Armaroli Annarita, Gnudi Saverio, Angelin Alessia, Grumati Paolo, Michelini Maria Elena, Franchella Andrea, Gualandi Francesca, Bertini Enrico, Maraldi Nadir Mario, Ferlini Alessandra, Bonaldo Paolo, Bernardi Paolo
Abstract excerpt
Six individuals with Ullrich congenital muscular dystrophy (UCMD) and mutations in the genes-encoding collagen VI, aging 5-9, received 3-5 mg/kg of cyclosporine A (CsA) daily for 1 to 3.2 years. The primary outcome measure was the muscle strength evaluated with a myometer and expressed as megalimbs. The megalimbs score showed significant improvement (P = 0.01) in 5 of the 6 patients. Motor function did not...
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