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.

After Intellia Therapeutics paused a pair of phase 3 trials for its CRISPR therapy in response to a liver safety signal, the ...

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 ...

BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks ...

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, ...

A research study recently published in the American Journal of Gastroenterology explains the role of pancreatic enzyme therapy (PERT) in reducing the frequency of acute pancreatitis (AP) in children ...

Dublin, Oct. 02, 2025 (GLOBE NEWSWIRE) -- The "Mucopolysaccharidosis Market - A Global and Regional Analysis: Focus on Treatment, Disease Type, Route of Administration, End User, Country, and Region - ...

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.

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 ...

Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ...