by David Hamlin | Sep 26, 2019 | News
Recognizing Pompe disease, a progressive, debilitating and often fatal neuromuscular disease caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), can be challenging, as its signs and symptoms are like those of other...
by David Hamlin | Mar 10, 2019 | News
A biotech company that conducts research and develops treatments for people living with rare diseases such as Pompe disease wants your help. They are recruiting Pompe patients and caregivers to take a short online survey about your knowledge and understanding of...
by David Hamlin | Sep 10, 2018 | News
Amicus Therapeutics Announces Regulatory and Clinical Updates for AT-GAA in Pompe Disease CRANBURY, N.J., Sept. 10, 2018 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel...
by David Hamlin | Aug 22, 2018 | News
STRIDE STUDY A Study in Subjects With LOPD Who Are Currently Being Treated With ERT VAL-1221 for Pompe Disease VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease neoGAA ...
