News

ERT Before Age 16 May Slow Classic Fabry Progression

Beginning enzyme replacement therapy (ERT) before age 16 is associated with reduced kidney and heart damage — relative to no treatment — in males with classic Fabry disease, according to a small study from the Netherlands. These early findings, concerning 10 years of treatment with Fabrazyme (agalsidase beta),…

Q&A With RARE-X Disease Data Platform Founder, Nicole Boice

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

Rare Disease Day Events Bring Awareness, Equity to Patients

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Fabry International Network Seeks Entries for FIN Award 2022

The Fabry International Network (FIN) is calling for global submissions for its annual FIN Award, which recognizes the efforts of patient organizations to educate and unify the Fabry disease community and to heighten awareness of the rare genetic lysosomal storage disorder. Projects must be initiated this year for…