Four cases of infantile-onset Pompe disease recently reported in Australia help shed light on some of the unique challenges care…
Articles by Alice Melao
The National Institutes of Health (NIH) has awarded EpiVax with a Small Business Innovation Research (SBIR) grant of $324,980.
Treatment with enzyme replacement therapy can help children with classic infantile Pompe disease reach adulthood. However, it fails to…
Amicus Therapeutics is recruiting participants for the fourth group of its ongoing Phase 1/2 clinical trial evaluating the company’s…
Duke Health researchers are planning to launch a Phase 1 trial to test the safety of a new investigational…