Click on the photo for a full description

There are millions of people worldwide with rare diseases who are in need of accurate diagnoses and access to appropriate treatments. Many people share their stories and opinions with me. This blog is meant to give them voice so that together we can continue the global crusade for orphan drugs to treat rare diseases.

Rare disease physicians

Note from Leslie Boyer

Message from Marybeth Krummenacker

Living with Cystinosis

message from Nancy Harris

Undiagnosed