First Duchenne Muscular Dystrophy Boy in Canada to Begin Drisapersen Redosing

Five years ago, 12-year-old Alex Chiabai was one…

Welcome to Scientific Curiosity.
I never expected for my very first question to get picked for an answer (see first question here), and then last Friday I was so excited to… Continue Reading
Resentment is an incredibly strong emotion.  Most of us will deny that we resent anything.  We might say we are angry, frustrated,… Continue Reading
Orphan Drugs Rare Disease Opportunity, Investors Eye
Orphan drugs don't have the potential patient numbers that cholesterol or diabetes drugs have, but that doesn't mean you should ignore them… Continue Reading
Emergencies: Are You Prepared?
Depending on the specific rare disease your family faces, having the right kind of emergency care can mean the difference in life or… Continue Reading
2010 Climb to Cure Duchenne – Pick Your Peak
Teams from around the country are climbing on June 19th or 20th. We want to get as many teams as possible to get out there and raise… Continue Reading
Medical Foods Equity Act and PKU
Act now to get your representative to sign on to the Medical Foods Equity Act (H.R. 4926) to support people with phenylketonuria (PKU) and… Continue Reading
Rare Disease Congressional Caucus Announced
John Crowley announced the Rare Disease Congressional Caucus this week. Leaders include Congressman Fred Upton and Congressman Joseph… Continue Reading

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