Full MIST2 dosing of intrapleural enzyme therapy varies widely across hospitals and is associated with a lower risk for ...

Tividenofusp alfa treatment showed reduction and normalization in key disease biomarkers, stabilization or improvement in clinical endpoints ...

Gold nanoparticles that cluster in response to T cell enzymes can predict cancer immunotherapy success days before tumors ...

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 ...

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 ...

Obesity is a global epidemic and a major cause of morbidity and mortality because it increases the risk for comorbidities, including heart disease and fatty liver disease (MASLD).

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

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 ...