Searching for a Cure For Duchenne – Patient Perspective Podcast #1

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In this first in a series of podcasts focused on rare disease patient advocates, we speak to Debra Miller, president and founder of Cure Duchenne. When Miller’s five-year-old son was diagnosed with Duchenne Muscular Dystrophy, she faced grim news. The progressive muscle loss caused by […]

Collaboration is key in rare disease research!

KTVU-TV 2, the leading evening news in the San Francisco Bay Area, today reported on Addi and Cassi Hempel, identical twins affected by a rare disease called Niemann-Pick Type C (NPC). NPC is fatal and degenerative cholesterol disease that is often called the “childhood Alzheimer’s.”  […]