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
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
Tell us about HemoShear – What progress have you made in inherited metabolic disorders? Q & A with HemoShear Brian Wamhoff, PhD, is Co-Founder and Head of Innovation at HemoShear Therapeutics, a biotechnology company developing drugs to treat metabolic disorders
Help us teach physicians about organic acidemias FACT! Teaching about metabolic diseases in medical school and residency programs is poor.FACT! Most patients live and die without a diagnosis being made, especially when the disease presents in adulthood.FACT! Patients cannot access effective therapies unless
MMA & PA Insight Study HemoShear Therapeutics HemoShear Therapeutics Receives FDA Fast Track and Rare Pediatric Disease Designations for HST5040 to Treat Methylmalonic Acidemia and Propionic Acidemia HemoShear Therapeutics Receives FDA Clearance of IND for Phase 2 Study of its
A new hepatic biomarker in MMA: Mice help patients Irini Manoli MD, PhD and Charles P. Venditti MD, PhD, National Human Genome Research Institute This article provides a basic summary of a paper we recently published entitled “FGF21 underlies a
