Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles...
Phoenix was born July 18th, 2003. I remember reading his APGAR test and the words of the neonatologist were “strong” however, I couldn’t tell if he was referring to his heart or his muscles. At any rate, he showed no sign of any serious problems other than the fact...
First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006 Enrollment of approximately 20 total study participants is ongoing PHILADELPHIA, February 1, 2021 (GLOBE NEWSWIRE)— Spark Therapeutics, a member of the Roche Group (SIX: RO,...
The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April 4, 2020 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...
Webinar Details Title: Informative Session on the Covid 19 Vaccine with Questions and Answers Date: Thursday, January 14, 2021 Speaker:Dr. Priya Kishnani Speaker Biography Dr. Priya Kishnani, MD Chief, Division of Medical Genetics, Department of Pediatrics; Professor...
Webinar Details Title: National Patient-Teacher Registry: A New Educational Concept Date: Wednesday, December 9, 2020 Speaker:Mark Korson, PhD, VMP Genetics, LLC, Director of Physician Support, Director of Education Webinar Overview:In general, medical students and...
Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe. To read the Pompe Program Update, please click here.
