好色先生

好色先生

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Abstract Details

GDF-15, lactate as well as clinical grading scale was improved by Sodium Pyruvate Therapy in Mitochondrial Myopathy
Child Neurology and Developmental Neurology
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012
Although many clinical trial have been conducted, no therapeutic drugs has been approved for mitochondrial disorders in the world.
Thirteen patients were selected from 74 mitochondrial patients registry by following selecting criteria; patients who fulfilled clinical criteria of either MELAS, Leigh or Kearns-Sayre syndrome, defined the respiratory chain enzyme defects, had known genetic abnormality, and had lactate > 2.5mmol/l. After written informed consent, Patients were administered 0.5 g/kg of SP TID and monitored lactate, pyruvate, L/P ratio, alanine, FGF-21 and GDF-15. They were also evaluated the clinical rating scale including JMDRS and NMDAS before, 3 months and 6 months after the administration of SP.
Thirteen patients include Leigh (PDHE1A) (1), 9 patients have Cardiomyopathy (2), MELAS (2), MELAS terminal stage (2), MELA (3) with an A3243G mutation, MELAS/Leigh overlapping with G13513A mutation (1), and Kearns Sayre syndrome with common deletion of mtDNA (2). Lactate, pyruvate and GDF-15 levels before, and 3 months after the administration of SP were 50.7 + 12.1 , 35.5 + 11.1 , 2.1 + 1.01, 1.4 + 0.75, and 3541.3 + 1960.4, 2845.2 + 1861.2 (mean + SD). Total score of section 1& 2 in JMDRS, and section 1-3 in NMDAS before, and 3 months after the administration of SP were 24.2 + 18.33, 21.2 + 18.11, and 57.2 + 43.76, 54.2 + 45.3 (mean + SD).
SP significantly improved the levels of lactate, and GDF-15 which was harmonized with the improvement of clinical rating scale observed in JMDRS and NMDAS. Considering that pyruvate therapy is a potential candidate for therapeutic choice for patients with mitochondrial diabetes.
Authors/Disclosures
Michio Hirano, MD, FAAN (Columbia University Medical Center) Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB Biopharma SRL. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Precision Biosciences. Dr. Hirano has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Biopharma SRL. Dr. Hirano has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apollo Communication. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Envision Communications. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for 好色先生. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Cure SMA. Dr. Hirano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Muscular Dystrophy Association. The institution of Dr. Hirano has received research support from UCB. The institution of Dr. Hirano has received research support from Cyclerion. Dr. Hirano has received research support from Reneo Pharmaceuticals. The institution of Dr. Hirano has received research support from Astellas. Dr. Hirano has received intellectual property interests from a discovery or technology relating to health care. Dr. Hirano has received intellectual property interests from a discovery or technology relating to health care. Dr. Hirano has received personal compensation in the range of $0-$499 for serving as a Study Section Reviewer with NIH. Dr. Hirano has a non-compensated relationship as a Research Advisory Board member with Muscular Dystrophy Association that is relevant to AAN interests or activities. Dr. Hirano has a non-compensated relationship as a Scientific and Medical Advisory Board member with United Mitochondrial Disease Foundation that is relevant to AAN interests or activities. Dr. Hirano has a non-compensated relationship as a Scientific Advisory Board member with Barth Syndrome Foundation that is relevant to AAN interests or activities.