Fishing for New Drugs for Propionic Acidemia

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

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What causes high ammonia and glycine in MMA?

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

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PA Study at the NIH

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

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MMA Study at the NIH

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)

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Tell Us About Moderna

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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Tell Us About LogicBio

Tell us about LogicBio Fred Chereau is the president and CEO of LogicBio Therapeutics, a biotechnology company in Cambridge, Mass., that is advancing an investigational therapy for methylmalonic acidemia(MMA). We sat down with him to talk about his work. Tell

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