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

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

Food and Drug Administration has placed a clinical hold on Intellia Therapeutics' two late-stage trials testing an ...

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, ...

Japan's Ministry of Health, Labor and Welfare (MHLW) has granted orphan regenerative medicine product designation to Kyowa ...

Shortly after taking reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

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 . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

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.

Preclinical study showed PDIA1 and PDIA5 protect cells from oxidative stress, maintain mitochondrial function, and are ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...