Prothena Shares Tumble as Company Discontinues AL Amyloidosis Candidate

Rare Daily Staff Prothena said it is discontinuing…

Advocate For Your Rare Disease or Foundation – Submit Your Story To Global Genes
We are looking for rare and genetic disease patient advocates to submit their stories of hope and inspiration to the Global Genes Project… Continue Reading
Six Year Old with Juvenile Metachromatic Leukodystrophy Is Mom’s Super Hero
Meet our super hero Peyton!  Peyton is six years old and is afflicted with Juvenile Metachromatic Leukodystrophy (MLD). Juvenile MLD is a… Continue Reading
Co-Pay Assistance Programs in Massachusetts – Why “Perpetuity” Provision (Senate Amendment 655.1) Will Not Help Patients
Both the House and the Senate have included in their FY 2013 budgets a provision that allows co-pay assistance programs to be used in… Continue Reading
A Lifestyle Change of Quitting Smoking Causes Behcet’s Disease Symptoms To Advance
My name is Tracey and 13 years ago Behcet’s Disease came into my life. Behçet’s disease (BEH-chets), sometimes called Behçet’s… Continue Reading
The Strange Tales of Ben Beesley – An Inspiring Children’s Book About Sanfilippo Disease
Matt McNeil is inspiring. Despite the fact that both of his children, Waverly and Oliver, share the same devastating genetic disease – MPS… Continue Reading
Accessing Rare Disease Data using the ODDT App available through iTunes
Upon receiving the news there would be an application I could install on my phone, which would not only give me access to Twitter and hot… Continue Reading
Death Without A Diagnosis – A Mother Describes The Moments of Losing Her Son at Age 5 From an Undiagnosed Rare Disease
Cal’s Story My son Cal had been battling an as yet undiagnosed disease for three years. And now at the tender age of five, he was… Continue Reading