Protalix BioTherapeutics (PLX) said on Friday that an expert panel of the European Medicines Agency (EMA), endorsed a new ...

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...

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.

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 ...

Studies of Hunter and Hurler syndromes on hold ...

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, ...

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.

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...