Lentiviral Gene Therapy for Pompe Disease

Lentiviral Gene Therapy for Pompe Disease

Oct 10, 2023 | PAST WEBINARS

Webinar Details Title: Lentiviral gene therapy for Pompe disease Date: Tuesday, October 10, 2023 Speaker:Dr. Pim Pijnappel, Associate Professor in Cell and Gene Therapy at the Erasmus MC University Medical Center, Rotterdam, the Netherlands Webinar Overview:The...
Spark Therapeutics Webinar: Trial Expectations

Spark Therapeutics Webinar: Trial Expectations

Sep 27, 2023 | PAST WEBINARS

Webinar Details Title: Spark Therapeutics Webinar: Trail Expectations – What to Expect Date: Wednesday, September 27, 2023 Speaker:Jacose Bell Webinar Overview:This presentation, developed by Spark Therapeutics, takes audience members through the introductory...
How to Manage LOPD Patients Diagnosed Through NBS

How to Manage LOPD Patients Diagnosed Through NBS

Aug 10, 2023 | PAST WEBINARS

Webinar Details Title: New insights into Pompe disease since the advent of NBS Date: Thursday, August 10, 2023 Speakers:Dr. Priya Kishnani, MDErin Huggins, MS, CGC Webinar Overview:An overview of the lessons learned from newborn screening for Pompe disease and its...

CALL to participate in IPA Project Every Move Counts

Aug 3, 2023 | NEWS, ARCHIVES

At International Pompe Day the International Pompe Association kicked off the Every Move Counts project. This project focuses on raising awareness of the importance of movement. We all have different abilities and levels of disability, but the important thing to...

Rare Artist Contest is now Open for Submissions

Aug 2, 2023 | NEWS, ARCHIVES

Enter July 23rd – August 31st at RareArtist.org The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals impacted by rare disease to tell their story through art. Now in year 13, the Rare Artist Annual Contest is focused on...