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.
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.
Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.
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.
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, a gene therapy biotech company, is on track to file an Investigational New Drug (IND) application in 2019 for Pompe disease.
Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapies
Focussing on Pompe, Fabry, CDKL5, Amicus Therapeutics begins a collaboration with the Gene Therapy Program at the Perelman School of Medicine at University of Pennsylvania.
Spark Therapeutics announced new preclinical data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease.