Blog
Audentes Therapeutics Update on Pompe and Other Programs
Audentes Therapeutics provides updates on their AAV-based genetic medicine program for serious rare neuromuscular diseases.
2019 CAP Annual General Meeting
The 2019 annual general meeting for Canadian Association of Pompe will be held by conference call on Sunday, May 26, 2019.
Amicus Therapeutics Presents Positive Preclinical Pompe Disease Gene Therapy Data
In what could eventually be very good news for Pompe patients, Amicus Therapeutics presents positive data from their preclinical gene therapy program for Pompe disease.
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.