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 ...

In early 2022, Connor started a new medication called Nexviazyme, but each treatment took 13 hours. This summer, he received ...

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.

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

Denali Therapeutics (($DNLI)) announced an update on their ongoing clinical study. Denali Therapeutics has launched an open-label extension study ...

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.

October 18, 2010 (Toronto, Ontario) — Enzyme replacement therapy using bone-targeted tissue-nonspecific alkaline phosphatase (TNSALP) is a promising treatment for children with hypophosphatasia, ...

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 ...

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.

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 ...