Blog
April 15, A Day of Reflection
April 15 is International Pompe Day. After losing too many patients the past few years, this year I’m filled with more questions than answers.
International Pompe Day – Moving On with Pompe!
The IPA believes that raising awareness about Pompe Disease is key, and for this year the theme that has been selected is “Moving On with Pompe”.
Audentes Therapeutics’ “Be Bold for Courageous Patients” Program to Support Patients Living with Rare Diseases
In recognition of Rare Disease Day, Audentes Therapeutics announces their new “Be Bold for Courageous Patients” program.
Amicus Therapeutics establishes a 75,000 sq. ft. Gene Therapy Center of Excellence
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.
Amicus Therapeutics Receives Breakthrough Therapy Designation for Pompe Treatment
Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.
Rare Disease Day 2019
February 28, 2019 will be the twelfth international Rare Disease Day coordinated by EURORDIS. On and around this day hundreds of patient organisations from countries and regions all over the world will hold awareness-raising activities.
Sarepta Therapeutics Enters into a Strategic Relationship with Aldevron in Support of Their Gene Therapy Program
Aldevron will supply plasmid DNA to fulfill Sarepta Therapeutics’s needs for its gene therapy clinical trials and commercial supply.
CAP Walks for Muscular Dystrophy in 2018
Canadian Association of Pompe (CAP) members, their friends and families walked to support Muscular Dystrophy Canada (MDC) in 2018, raising over $13k.
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study of AT-GAA in Patients with Pompe Disease
Amicus Therapeutics has announced that the first patient has been dosed in their Phase 3 study of AT-GAA for patients with Pompe disease.
Audentes Therapeutics Pompe Program Update
Audentes Therapeutics, a gene therapy biotech company, is on track to file an Investigational New Drug (IND) application in 2019 for Pompe disease.