The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More research. This
November 7, 2023
Ask Bio Webinar: Development of Gene Therapy for Pompe Disease
Webinar Details Title: Development of Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More: Recent History Date: Tuesday, November 7, 2023 Speaker:Dr. Dwight Koeberl
Webinar Details Title: Defining Central Nervous System Abnormalities in Infantile and Late-Onset Pompe DiseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More Patients Date: Tuesday, October 17, 2023
October 10, 2023
Lentiviral Gene Therapy for Pompe Disease
Webinar Details Title: Lentiviral gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Date: Tuesday, October 10, 2023 Speaker:Dr. Pim Pijnappel, Associate Professor in
October 4, 2023
Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease
PHILADELPHIA, Sept. 28, 2023 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration
September 27, 2023
Spark Therapeutics Webinar: Trial Expectations
Webinar Details Title: Spark Therapeutics Webinar: Trail Expectations – What to Expect Date: Wednesday, September 27, 2023 Speaker:Jacose Bell Webinar
August 10, 2023
How to Manage LOPD Patients Diagnosed Through NBS
Webinar Details Title: New insights into Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More since the advent of NBS Date: Thursday, August 10, 2023 Speakers:Dr. Priya
August 3, 2023
CALL to participate in IPA Project Every Move Counts
At International Pompe Day the International Pompe Association kicked off the Every Move Counts project. This project focuses on raising
August 2, 2023
2024 Acid Maltase Deficiency Association (AMDA)/International Pompe Association (IPA) Conference date has been set!
Dear Pompe Warriors and Patient Community, We are excited to announce the upcoming 2024 Acid Maltase Deficiency Associate – International
August 2, 2023
Rare Artist Contest is now Open for Submissions
Enter July 23rd – August 31st at RareArtist.org The Rare Artist Program was established in 2010 to exhibit the unique