Recent Posts
Amicus Remains Hopeful about AT-GAA Despite Mixed Results from Phase 3 Study
Amicus Therapeutics recently announced topline results of its phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD).
Spark Therapeutics Has Begun a Phase 1/2 Gene Therapy Study for Late-Onset Pompe Disease
The Spark Therapeutics phase 1/2 gene therapy trial testing SPK-3006 to treat late onset Pompe disease has officially started with the first person being given their first (and theoretically only) dose.
Amicus Therapeutics Has Begun the FDA Submission for Their Pompe Drug, AT-GAA
Amicus Therapeutics has announced they have begun a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for their drug combination, AT-GAA, to treat late onset Pompe disease (LOPD).
New Review Article Highlights Latest Developments in Pompe Disease Research
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
New ERT For Late-onset Pompe Disease Shows Promise
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
What is Pompe disease?
Pompe disease is one of about 50 Lysosomal Storage Disorders. Pompe patients are deficient in the critical enzyme acid alpha-glucosidase.
Connections
Ways to connect with other patients & families and social media opportunities to stay informed.
New Patients and Families
Welcome to the wonderful world of rare disease. It’s a much different world than it was a few years ago. There’s so much information out there, and there are places to turn for support and friendship. With social media, especially Facebook and Twitter, there are answers.
There are patient advocacy groups all over the world, of which CAP is one of them.
New Brunswick, 2014
Out of a stormy cloud comes a whole bunch o’ crazy people waving their hands… at the 2014 Fredericton Walk for Muscular Dystrophy. We were fortunate to gather in New Brunswick for our 2014 CAP Conference and were humbled to be presented with the Kaitlyn Hatchard Award with Annette Sebey.
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Mailing Address
112-3201 Wilson St
Penticton, BC,
Canada
V2A 8J3