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 ...

A Denali Therapeutics drug for the rare enzyme deficiency Hunter syndrome is still in pivotal testing, but the company has guidance from the FDA on a pathway to get this therapy to the market sooner.

BioMarin Pharmaceutical is building up its pipeline with the acquisition of Inozyme Pharma in a $270 million deal centered on an enzyme replacement therapy that could become the first FDA-approved ...

The FDA approved the enzyme replacement therapy Adzynma for the prophylactic or on-demand treatment of adults and pediatric patients with congenital thrombotic thrombocytopenic purpura, a rare blood ...

VIENNA, Austria — The appropriate dose of pancreatic enzyme replacement therapy (PERT) for exocrine pancreatic insufficiency (EPI) depends on the root cause of the insufficiency, according to results ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week. It's been a ...