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Recent Posts
- Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds
- LOPD Adults in China Cite Poor Quality of Life
- Celebrating All of My Child’s Milestones
- Nominations Open for 2022 Eurordis Black Pearl Awards
- Study Stresses Importance of Early Treatment for Different Mutations
- My Son Has Used Several Types of Orthotic Braces Due to Pompe Disease
- Staying in the NICU After My Son’s Birth Helped With Postpartum Emotions
- New GAA Mutations ID’d as Cause of Late-onset Disease
- Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years
- Different Types of Mutations Appear to Predict Pompe Subtypes