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 ...
by United Pompe | Jul 26, 2018 | News
Higher Lumizyme Dose May Help Prevent Pompe Progression in Infants, Case Report Suggests JULY 18, 2018BY ALICE MELAOIN NEWS. Treatment with Lumizyme(alglucosidase alfa) at double the approved dose may help prevent the rapid decline of respiratory...
by United Pompe | Jul 10, 2018 | News
Amicus Therapeutics Strengthens Biologics Business Strategy
