Blog
Nexviazyme Meets All Endpoints in Phase 3 Study for Infantile-Onset Pompe Disease
Sanofi has announced positive results from the Baby-COMET phase 3 study, which evaluated Nexviazyme (avalglucosidase alfa) in infants with infantile-onset Pompe disease (IOPD). The study met its primary endpoint and all secondary endpoints, and Sanofi intends to use...
First Participant Dosed in Phase 1/2 Gene Therapy Trial for Late-Onset Pompe Disease
AskBio has announced that the first participant has been dosed in a phase 1/2 clinical trial evaluating AB-1009, an investigational gene therapy for adults with late-onset Pompe disease (LOPD). AB-1009 is designed to address the underlying cause of Pompe disease by...
First Patients Enrolled in Phase 2 Trial of SRT for Late-Onset Pompe Disease
Shionogi has announced the enrollment of the first patients in a global phase 2 clinical trial evaluating S-606001, an investigational oral therapy for adults with late-onset Pompe disease (LOPD). S-606001 is a substrate reduction therapy (SRT) designed to reduce...
Aro Biotherapeutics reports positive phase 1b results for ABX1100
Aro Biotherapeutics has reported positive topline results from its phase 1b clinical trial of ABX1100, an investigational therapy for adults with late-onset Pompe disease (LOPD). ABX1100 is a substrate reduction therapy (SRT) designed to reduce glycogen production in...
2025 Pompe Empowerment Workshop : Vancouver
This Pompe Empowerment Workshop welcomes patients and their family from BC to MB, for networking, learning and sharing.
Welcome Victoria Radke!
Please join us in welcoming Victoria Radke as our Junior Executive Director! Victoria has 6 years of post-secondary education with a Bachelor’s Degree in Psychology and a Post-Degree Diploma in Human Resources and Leadership. She has been an Associate Editor at Zeal...
Aro Biotherapeutics starts Phase 1b Clinical Trial for Substrate Reduction Therapy ABX1100
Aro Biotherapeutics has launched a Phase 1b clinical trial for ABX1100, a substrate reduction therapy (SRT).
2024 Pompe Empowerment Workshop : Toronto
This Pompe Empowerment Workshop welcomes patients and their family from Ontario or the Maritimes, for networking, learning and sharing.
Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001
This Substrate Reduction Therapy drug could be the first pill taken by mouth to treat Pompe disease.
#Pies4Pompe Challenge
The #Pies4Pompe Challenge is an awareness campaign hosted by Canadian Association of Pompe and driven by community and public participation.