New Research Provides Insight Into How Propionic Acidemia Changes Liver Metabolism Guo-Fang Zhang, PhD, Duke University Propionic acidemia (PA) is caused by deficiency of an enzyme called propionyl-CoA carboxylase (PCC), which is needed to break down propionyl-CoA. When PCC does
Update on The Rapid Blood Ammonia Testing Instrument Thomas Veltman, Ph.D.President and CTOAza Technology, Inc.tv@azatechnology.com Aza Technology has made significant progress towards the commercialization of our point-of-care Ammonia Monitoring Device (AMD). In our previous update, we informed readers that we
Fishing for New Drugs for Propionic Acidemia BY: VIRGINIA GINOCCHIONICOLA BRUNETTI-PIERRI, MD, FACMGASSOCIATE INVESTIGATORTELETHON INSTITUTE OF GENETICSAND MEDICINE VIA CAMPI FLEGREI 3480078 POZZUOLI (NAPOLI), ITALYASSOCIATE PROFESSORDEPARTMENT OF TRANSLATIONAL MEDICINEFEDERICO II UNIVERSITY OF NAPLES In the last decades careful clinical and
What causes high ammonia and glycine in MMA? New insight into disease mechanisms from mouse and human tissues and a possible new treatment. MMA produces unique protein modifications that contribute to disease Dr. Charles Venditti’s lab at the National Institutes
Point-of-Care Ammonia Monitoring from a Single Drop of Blood Thomas Veltman, Ph.D., Aza Technology, Inc.tv@azatechnology.com In an earlier article in this newsletter, I described my efforts, along with a team of other researchers, towards producing a point-of-care ammonia meter with
PA Study at the NIH Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia Dr. Chuck Venditti and Dr. Oleg Shchelochkov seek Propionic Acidemia patients for their study. Hi – We are happy to announce that the NIH has
MMA Study at the NIH The NIH has been recruiting MMA patients to their study since 2004. Below are links that describes the study and how to sign up. Clinical Study Links: Clinical and Basic Investigations of Methylmalonic Acidemia (MMA)
A Novel Gene Medicine Approach for the Treatment of Rare Early Onset Diseases LogicBio Therapeutics, Cambridge, MA The field of gene therapy has experienced a renaissance in recent years, having rebounded from the clinical setbacks of decades past. The
A novel messenger RNA therapeutic approach for the treatment of methylmalonic and propionic acidemias Moderna Therapeutics, Cambridge, MA MMA produces unique protein modifications that contribute to disease Moderna Therapeutics is a seven-year old company focused exclusively on developing medicines and
Tell us about Moderna Kelly Lindert, MD, is the Head of Metabolic Rare Diseases Clinical Development at Moderna, a biotechnology company in Cambridge, Mass. that is working to develop messenger RNA (mRNA) therapeutics for a variety of areas, including rare
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