2019 AMDA/IPA International Conference
The AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from October 25-27, 2019. Please click here for the 2019 Conference Registration Form. Each family, speaker, patient organization representative, industry...2019 PCMA’s Pull For Pompe!
The AMDA is excited to announce that the 2019 PCMA’s Pull for Pompe will take place on Saturday, April 13th at the National Shooting Complex in San Antonio, Texas! This is the 9th Annual Pull for Pompe hosted by the PCMA. The Pull for Pompe clay shooting event is...Press Release: Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe Disease Global Study to Assess AT-GAA Compared to Standard-of-Care Enzyme Replacement Therapy (ERT)A treatment that replaces the missing enzyme through...Spark Therapeutics Gene Therapy Research Webinar
Spark Therapeutics Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. Research Webinar Download an audio only...Press Release: Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate Data from IND-enabling studies in three species support moving SPK-3006 into the clinic in 2019 PHILADELPHIA, Oct. 08, 2018—Spark Therapeutics, a fully integrated, commercial...