Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ...

"There are three digestive enzymes made by your pancreas," says Mary Vouyiouklis Kellis, MD, a specialist in the department of endocrinology, diabetes and metabolism at the Cleveland Clinic. "Most ...

Purpose: Infantile Pompe disease is caused by deficiency of lysosomal acid α-glucosidase. Trials with recombinant human acid α-glucosidase enzyme replacement therapy (ERT) show a decrease in left ...

Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected ...

A new study led by University of Pittsburgh and UPMC Hillman Cancer Center researchers shows that an enzyme called PARP1 is involved in repair of telomeres, the lengths of DNA that protect the tips of ...

As the name implies, a rare disease is an illness that affects far fewer people than more common conditions, like breast cancer or heart disease. Today, however, 7,000 rare conditions have been ...

Stem cells transplanted in utero (green) engrafted into fetal mouse brain tissue. Image credit: Q-H Nguyen/MacKenzie lab. Administering stem cell or enzyme therapy in utero may be a path to ...

Following the completion of this acquisition, BioMarin will add Inozyme’s lead asset, INZ-701, to its pipeline. This investigational enzyme replacement therapy (ERT ...