The Importance of Newborn Screening

The Importance of Newborn Screening

Read Gianna’s story and how it changed the course of her life. Newborn screening gave Gianna a head start in her fight against MPS I. Today marks the 1-year anniversary of Gianna's bone marrow transplant. So amazing!

2021 National Family Conference and Gala

2021 National Family Conference and Gala

Our bi-annual conferences give families affected by MPS & related diseases an opportunity to learn about new research, treatment and care, and to share information and experiences with other affected individuals and families. The event was enjoyed virtually on...

Resilience in Rare Diagnosis

Resilience in Rare Diagnosis

As part of the Virtual Education and Connection Series, listen to our very special speakers talk about their unique and resilient stories regarding a rare disease diagnosis. https://youtu.be/l5ybp_PEtjM

Christina Gentle at the 2018 RARE Gala

Christina Gentle at the 2018 RARE Gala

Written and presented by Christina Gentle at the 2018 RARE Gala "Hi everyone, thanks for being here tonight. I’m Christina, Jessica Gentle’s sister. If you were at the Gala last year you might remember the great speech she gave about what it’s like to live with MPS 1,...