HemoShear Therapeutics Making Progress Toward Potential Treatment for OA Diseases HemoShear Therapeutics We all greatly desire new treatments to improve the quality of life for people with organic acidemias. In order for progress to be made, scientists need to better
What We Know About Ammonia In Organic Acidemias by: Nicolas Ah Mew, MD Many families and doctors who care for individuals with organic acidemias are very familiar with the fact that blood ammonia levels can be elevated during illness and
Renal Complications in Propionic Acidemia: Not as Rare as Previously Suspected Oleg A. Shchelochkov, MD, and Charles Venditti, MD, PhD Propionic acidemia (PA) is a serious metabolic disease which can impact many organs. For example, it has been known for
Gene Therapy Future for Propionic Acidemia When Charlize underwent her life-saving liver transplant, mum Julie made sure the cells went to the Gene Therapy Unit at Children’s Medical Research Institute (CMRI) in Sydney which is led by Professor Ian Alexander.
First Site Open in a Phase 1/2 Study of an Investigational mRNA Medicine for MMA Patients Messenger RNA (mRNA) plays a critical role in human biology, providing a set of instructions to cells in the body to make proteins. Moderna
Health-Related Quality of Life in Children and Families Affected by Methylmalonic Acidemia Knowledge of the psychosocial impact of methylmalonic acidemia (MMA) on children and families is necessary in planning counseling and therapeutic intervention. Although clinical outcomes of MMA have been
Autism & Metabolism STEPHEN G. KAHLER, MDPROFESSOR, DIVISION OF GENETICS AND METABOLISM DEPARTMENT OF PEDIATRICSUNIVERSITY OF ARKANSAS FOR MEDICAL SCIENCES LITTLE ROCK, ARKAHLERSTEPHENG@UAMS.EDU This essay is based on a talk I intended to give at the 2016 FOD-OAA meeting in
Liver Transplantation for Propionic and Methylmalonic Acidemias Jerry Vockley, M.D., Ph.D. Professor of Pediatrics, University of Pittsburgh School of MedicineProfessor of Human Genetics, University of Pittsburgh Graduate School of Public HealthChief of Medical Genetics, Children’s Hospital of Pittsburgh Liver transplantation
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