Aug 1, 2024 | ARCHIVES, NEWS
We are excited to announce that the topic of our first “Talking With Your Pompe Peeps” session is “The Elementary School Experience: Building Success for IOPD/LOPD Children Through Collaboration and Partnership”! Join us August 16, 2024 at 1 PM...
Jun 23, 2024 | ARCHIVES, NEWS
University of California – San Francisco (UCSF) is excited to announce the rebranding of their phase 1 clinical trail of In Utero Enzyme Replacement Therapy as the PEARL Trial-PrEnAtan enzyme Rreplacement for Llysosomal storage diseases. “We have rebranded...
Feb 26, 2024 | PAST WEBINARS
Webinar Details Title: Evaluating gene therapies for Pompe disease – clinical trial goals and assessments Date: Monday, February 26, 2024 Speaker:Jordi Díaz-Manera, MD, PhDChristine S. BrownMark Walzer, PhD Webinar Overview:This Astellas Gene Therapies...
Feb 10, 2024 | ARCHIVES, EDITORIALS
By: IPA posted on: May 27, 2019 The IPA has authored an article titled “The role of patient advocacy organizations in shaping medical research: the Pompe model,” and we are excited to announce that it has been accepted for publication. The article focuses on...
Jan 29, 2024 | NEWS, ARCHIVES
The AMDA is excited to announce that the 13th Annual PCMA’s Pull for Pompe will take place on Saturday, April 27, 2024 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
