Early UniQure Fabry trial shows lasting enzyme boost, safety watch

uniQure N.V. QURE on Friday announced updated preliminary safety and exploratory efficacy data from 11 patients in its Phase ...
News Medical

New gene therapy shows promise for lifelong treatment of hypophosphatasia

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week. It's been a ...
Business Wire

ATUM Partners with Anagram Therapeutics to Extend Collaboration to Develop Orally Delivered Enzyme Therapies for Malabsorption and Nutrient Metabolism Disorders

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 ...
Nasdaq

Denali Therapeutics Announces Presentations on Its Investigational Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapies at the Upcoming 2024 WORLDSymposium™

MPS II, also called Hunter syndrome, is a rare genetic disease that affects over 2,000 individuals, primarily males, world-wide, and leads to behavioral, cognitive, and physical symptoms ultimately ...