Recent Posts
AVROBIO to Explore Strategic Alternatives
One of a handful of gene therapy companies with pipelines that include Pompe disease. Today AVROBIO announced a halt to their programs.
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union
Amicus Therapeutics, announces the approval of Opfolda (miglustat) capsules, an enzyme stabilizer of cipaglucosidase alfa, by the European Commission for the treatment of Pompe disease.
International Pompe Day 2022
Pompe Day is wrapping up soon for another year. It’s important to point out that for Pompe families, Pompe Day is 365 days long.
Rare Disease Day 2022
We still live in a world where there are over 300 million people living with over 6,000 rare diseases. That’s enough to populate the 3rd largest country in the world. We have the tools to diagnose them quicker, but we aren’t doing it.
Maze Therapeutics Starting Phase I Clinical Trial with MZE001, A Substrate Reduction Therapy
Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for 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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112-3201 Wilson St
Penticton, BC,
Canada
V2A 8J3