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.
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.
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.
Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for Pompe disease.
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.
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.
112-3201 Wilson St