A Phase II Pilot Study to Assess Safety and Efficacy of Sodium Valproate in Adults with McArdle Disease

Update Il y a 4 ans
Reference: EUCTR2012-002933-12

Woman and Man

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Extract

To determine whether patients taking a medicine called sodium valproate can improve muscle function expressed by an increased number of normal muscle fibres on muscle biopsy.


Inclusion criteria

  • McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdomyolysis which can cause acute renal failure and from middle age muscle wasting and weakness. Affected patients are unable to produce lactate during ischaemic exercise due to a congenital lack of the enzyme muscle glycogen phosphorylase which is essential for glycogen metabolism

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