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

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.

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

BioMarin Pharmaceutical is building up its pipeline with the acquisition of Inozyme Pharma in a $270 million deal centered on an enzyme replacement therapy that could become the first FDA-approved ...

Two years ago, we shared the story of a 12-year-old boy from Lubec who made an eight-hour trip to Boston every other week for treatments that would help him live longer.Connor Haskins lives with a ...

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.

Dr. Drucy Borowitz helped turn cystic fibrosis from a deadly childhood illness into a treatable condition, giving families ...

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 - ...

Research into gene therapies began more than 40 years ago, but the first FDA approval in 2017 ignited a fresh wave of interest from investors and drug companies who see the technology’s transformative ...

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 ...

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.

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 ...