Scientists have successfully restored hearing in animal models of ENPP1 deficiency, a genetic disease in which individuals lack an enzyme essential for bone and blood vessel health. The rare disorder ...

JCR announced that IZCARGO, an enzyme replacement therapy indicated for Hunter syndrome, received marketing authorization in the United Arab Emirates.

Scientists' discovery of a molecular "switch" that activates an energy-burning pathway in mice has the potential to lead to new treatments for bone disease. The study, published in Nature, sheds new ...

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 ...

Hypophosphatasia (HPP) is an inherited metabolic disorder characterised by deficient activity of tissue non-specific alkaline phosphatase (TNAP), leading to impaired bone and dental mineralisation.

Mucopolysaccharidosis type 1, also known as Hurler syndrome, is a rare but life-threatening genetic condition. Babies born with this inherited disorder do not produce an enzyme needed to break down ...

(RTTNews) - uniQure N.V. (QURE), a gene therapy company developing treatments for severe genetic diseases, announced updated preliminary Phase 1/2a data for AMT-191, its investigational AAV gene ...

Zenpep (pancrelipase) is an enzyme replacement therapy made from pig pancreas enzymes. It’s prescribed for exocrine pancreatic insufficiency when your body can’t naturally make enough digestive ...