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.

Orsini, a provider of rare disease pharmacy solutions, is the exclusive specialty pharmacy partner for Denali Therapeutics' AVLAYAH, an FDA-approved enzyme replacement therapy ...

In the United States about 5%-8% of the population is affected by an autoimmune disorder. Other reports, however, estimate a higher prevalence of various types of autoimmune disorders due to complex ...

Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...

Cancer cells have ways to hide from the immune system, but there are some unique features on these dangerous cells that ...

Enzyme replacement therapy (ERT), which provides a synthetic version of missing GAA enzyme, is the primary treatment for this condition. It’s not a cure, but it can address symptoms for people in all ...

Most gene-editing tools cut DNA wherever they’re pointed, with no way to tell a tumor cell from a healthy one. A study ...

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 ...

Please provide your email address to receive an email when new articles are posted on . A phase 3 trial evaluating intrapleural enzyme therapy vs. video-assisted thoracoscopic surgery is feasible.

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 ...

Researchers from Drexel University’s College of Arts and Sciences and College of Medicine have found a potential new therapeutic target for Alzheimer’s disease. Expanding on their previous research on ...