Kearns-Sayre syndrome

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…

Early Folinic Acid Supplementation Improves Symptoms of Kearns-Sayre Syndrome

If patients with Kearns-Sayre syndrome commonly are deficient in folate, logic would presume folinic acid therapy may benefit their symptoms. A team from Spain tested this theory in the article “Follow-up of Folinic Acid Supplementation for Patients with Cerebral Folate Deficiency and Kearns-Sayre Syndrome,” which was…

Procedure Corrects Kearns-Sayre Syndrome Eyelid Drooping

Chronic progressive external ophthalmoplegia (CPEO) is the most common characteristic of Kearns-Sayre syndrome, which is one member of the family of mitochondrial myopathies. Patients with CPEO may benefit from bilateral fascia lata lid suspension, a procedure explored by researchers in Brazil and Italy to correct blepharoptosis, or severe drooping of…

Novel Therapy for Multiple Mitochondrial Diseases Now in Clinical Trials

Edison Pharmaceuticals is actively engaged in a number of phase 2 clinical trials investigating their drug candidate EPI-743 in a variety of mitochondrial respiratory chain diseases. The studies range from the more general “EPI-743 for Mitochondrial Respiratory Chain Diseases” to the more…