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.
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BioMarin Beats on Q3 Earnings, Seeks to Divest Hemophilia Gene Therapy
BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks ...
Ottawa, Sept. 24, 2025 (GLOBE NEWSWIRE) -- The global enzyme replacement therapy market size was valued at USD 10.4 billion in 2024 and is predicted to hit around USD 24.71 billion by 2034, rising at ...
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 - ...
Children with Hunter Syndrome lack a certain enzyme in their blood. The Food and Drug Administration just approved an enzyme-replacement therapy for these patients. It had its start at UNC Hospitals.
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 ...
In early 2022, Connor started a new medication called Nexviazyme, but each treatment took 13 hours. This summer, he received ...
uniQure N.V. (NASDAQ:QURE) on Friday released initial safety and exploratory efficacy data from the first cohort of its Phase 1/2a trial of AMT-191, an investigational gene therapy for Fabry disease.
In early trial data, INZ-701 was associated with several clinical benefits including a reduction or stabilization of carotid intima-media thickness. The Food and Drug Administration (FDA) has granted ...
October 18, 2010 (Toronto, Ontario) — Enzyme replacement therapy using bone-targeted tissue-nonspecific alkaline phosphatase (TNSALP) is a promising treatment for children with hypophosphatasia, ...
Pancreatic exocrine insufficiency (PEI) is a condition where the pancreas does not produce enough digestive enzymes, leading to difficulties in digesting food and absorbing nutrients. This condition ...
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