Progressive external ophthalmoplegia

Peripheral Neuropathy May Indicate Mitochondrial Myopathy

A simple blood or urine test, rather than an invasive muscle biopsy, may be all that is needed to diagnose mitochondrial disease in patients with peripheral neuropathy (muscle weakness or a pricking sensation in the extremities) and progressive external…

Raptor Pharmaceuticals’ Experimental Therapy in Phase 2 / 3 Trial for Leigh Syndrome

Raptor Pharmaceuticals Inc. is looking to bring relief to a wide variety of young patients with inherited mitochondrial disease through its therapeutic called RP103 (cysteamine bitartrate delayed-release capsules). A Phase 2/3 clinical trial is currently recruiting and treating patients with diseases resulting from nuclear or mitochondrial DNA mutations, which include…