Cyclosporine Efficacy with Ullrich CMD

Dr. Paolo Bernardi, Italy, presented data showing cyclosporine efficacy in the treatment of 6 patients with Ullrich CMD. Muscle biopsies prior to treatment compared with biopsies one month into treatment showed muscle cell regeneration. Greater effect in children than adults.  Mitochondrial Summit Meeting Phoenix, Arizona, cosponsored by FARA and MDA. 

Merlini L, AngelinA, Bernardi P, et al. Cyclosporin A corrects mitochondrial dysfunction and muscle apoptosis in patients with collagen VI myopathies. PNAS 2008 April 1:105 (13):5225-9.

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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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