Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

Rare disease drug developer BioMarin Pharmaceutical is buying its way to revenue growth with a $4.8 billion deal for Amicus Therapeutics, a company whose two commercialized products each offer ...

At the mRNA Health conference in Berlin, enginzyme and AGC Inc. presented a scalable process to produce a key mRNA vaccine and therapy ingredient, N1-methylpseudouridine-5'-triphosphate (m¹ΨTP).

Pancreatic enzyme replacement therapy (PERT) is the gold standard treatment for exocrine pancreatic insufficiency (EPI). It creates the enzymes your pancreas can’t make using digestive enzymes. PERT ...

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for ...