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.

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

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

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

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.

NEWARK, Calif.--(BUSINESS WIRE)--ATUM, a global specialist and industry leader in bioengineering solutions, today announced an expanded partnership with Anagram Therapeutics Inc., a clinical-stage ...

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 - ...

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 ...

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 ...

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.

Citizens JMP upgraded Spruce Biosciences (SPRB) to Outperform from Market Perform with a $254 price target The firm cites the company’s ...