This year, for International Pompe Day, we’re launching Portraits of Pompe. This is an ongoing curation of stories and photos from Pompe patients and families. Focusing first on patient portraits here in Canada, we’ll be expanding this program to include portraits from around the world. Our hope is that these portraits show how we are just like any other family. We have hopes and dreams and we want the very best for our kids.
- Amicus Remains Hopeful about AT-GAA Despite Mixed Results from Phase 3 Study
- Cheryl Wong Po Foo
- Spark Therapeutics Has Begun a Phase 1/2 Gene Therapy Study for Late-Onset Pompe Disease
- Amicus Therapeutics Has Begun the FDA Submission for Their Pompe Drug, AT-GAA
- New Review Article Highlights Latest Developments in Pompe Disease Research
advocacy awards Awareness business CAP chaperone clinical trials compassion CORD COVID-19 eLearning Emma Rooney ERT Extraordinary Measures Fabry FDA fundraising Gaucher gene therapy getting treated grants guest blogger home infusions IND Int'l Pompe Day IPA late-onset law living with Pompe meetings Muscular Dystrophy Canada MyNormal newborn screening NIH Orphan Drug Framework Orphanet politics portraits pre-clinical preclinical pregnancy privacy Rare Disease Rare Disease Day video