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

Successful Gene Therapy Treatment and Redosing in an Animal Model of Methylmalonic Acidemia Petr O. Ilyinskii and Takashi Kei KishimotoSelecta Biosciences, Watertown, MA Gene therapy is making a difference in the lives of patients with previously untreatable genetic diseases. Most of

New Drug Relieves TCA Cycle Block In Patients With PA And MMA by: Jerry Vockley, M.D., PH.D. UNIVERSITY OF PITTSBURGHCLEVELAND FAMILY PROFESSOR OF PEDIATRIC RESEARCHPROFESSOR OF HUMAN GENETICS, CHILDREN’S HOSPITAL OF PITTSBURGHCHIEF OF MEDICAL GENETICS DIRECTOR OF THE CENTER FOR

Current Research Articles on Propionic Acidemia Major Breakthrough on Propionic Acidemia Research PaVe-GT: Paving the Way for Rare Disease Gene Therapies Adeno-associated virus serotype 8 (AAV8) Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia New Drug Relieves

​Effective Gene Therapy for Propionic Acidemia in Mice: One Organic Acidemia Helps Another Randy J. Chandler, Nuria Carrillo-Carrasco, and Charles P. Venditti  Organic Acid Research Section, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD  Email:venditti@mail.nih.gov For many

Red Blood Cell Metabolism in Patients withPropionic Acidemia Propionic acidemia (PA) is a rare, autosomal recessive disorder engendered by a dysfunctional propionyl-CoA carboxylase (PCC) enzyme [1]. PA has an estimated incidence of 1:100,000 live births in the general population, and