Centyrin-siRNA Conjugates for the Treatment of Pompe Disease in a new Clinical Trial
Learn about a Phase 1b clinical trial for ABX1100, a substrate reduction therapy (SRT), which aims to slow disease progression.
Emerging From the Pandemic: Pompe Perspectives
Dr Aneal Khan, Alexandra Butler, Sherry Simo and Lochlan Smyth will recap and look forward to emerging from the pandemic.
The Genetic Non-Discrimination Act
Persons with genetic conditions cannot be asked by their employers, or any other agency, to disclose that information.
Virtual Town Hall: Being Rare in a Pandemic
A Q&A with Dr Aneal Khan, MSc, MD, FRCPC, FCCMG; Professor of Medical Genetics and Pediatrics, University of Calgary, Alberta Children’s Hospital.