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

Purpose: Enzyme replacement therapy (ERT) is a promising therapeutic intervention for lysosomal storage diseases. Posttranslationally engineered human β ...

AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. For more information, ...

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 ...

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 ...

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 ...

A large team of researchers led by Wouter Karthaus, head of the Endocrine Therapy Resistance and Molecular Genetics Lab at ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

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