30Dec

Why Rare Disease Companies Need the Pediatric Rare Disease Priority Review Voucher

Why Rare Disease Companies Need the Pediatric Rare Disease Priority Review Voucher -Originally published by the Rare Disease Report If there was a genetic lottery and you were picking out diseases, you would hope that you did not get 1 of the 7000 rare diseases – especially rare pediatric diseases. And the reason is simple; […]

11Nov

This Will Be Our Year, We Have Only Just Begun

This winter we will be launching our Giving Tuesday Campaign through Crowdrise Giving Tower.  Our theme is “This Will Be Our Year-We’ve Only Just Begun”.  Inspired by the song “This Will Be Our Year” covered by OK GO. (Click to listen) The idea behind Giving Tuesday is simple; on Black Friday we shop, on Cyber Monday we shop some more, on […]

22Sep

Patients Can’t Wait

Patients Can’t Wait Today, patient advocates from across the country will unite in a day of action to urge the Senate to make medical innovation a top priority.  In July, the House passed historic legislation, called the 21st Century Cures Act, by a wide bipartisan margin (344-77!!!).  The bill contains vital provisions that benefit the […]

14Aug

The Quest To Find The Patients For A Rare Disease

The Quest To Find The Patients For Sanfilippo Syndrome Type D or MPS III D For major diseases like diabetes, hypertension and cancer there are literally millions of patients, you probably know one or more person with one of these diseases, you may even have one of them. But when it comes to a rare […]

Latest Videos

Jonny Lee Miller – Running For A Sanfilippo Cure

Elementary star Jonny Lee Miller is running 50 miles to raise money to cure a rare and fatal disease called Sanfilippo. Hear him talk about his training and why he is running. Catch Elementary Thursdays at 10/9c. Only CBS