Our 8-year-old daughter Anaïs has Morquio syndrome. Each additional year spent at her side makes us proud as parents. She is wise and mature beyond her years, with strengths like perseverance, determination, and living each day fully without fear. But as time passes,...
Our daughter Josephine was diagnosed with MPS 1 Hurler Syndrome on her first birthday. After a stem cell transplant at 18 months, she has been doing relatively well. However, she recently became self-conscious about looking and moving differently. She stared to...
When my son Elovic was diagnosed with MPS at age 3, I was in shock. It felt like my world was tearing at the seams. I was faced with so many unknowns and I wasn’t certain how to deal with everything that was being sent my way. That’s when I reached out to the Canadian...
November 8, 2023 Dear MPS I community, This afternoon REGENXBIO Press Release 8Nov2023, we announced a corporate restructuring that impacts our development of potential AAV gene therapies for MPS I. We are disappointed to share that REGENXBIO will no longer be moving...
Caring for a child with a rare disease like MPS can feel isolating at times – but you don’t have to go it alone. We’re bringing together a compassionate group of parents and caregivers who have been there and can offer a listening ear, share advice,...
