Bunco, Bid, & Boogie for Jonah’s Just Begun
Your are cordially invited to an evening filled with food, friends, music and auction.
To be held Friday, May 20th from 6:30-10:30 PM PDT
At Bella Via, Sherwood, OR
Enjoy all that this event has to […]
Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest contributors, including patients, stakeholders, innovators and others, to share their perspectives and point of view. Views represented here may not be those of PhRMA, though they are […]
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 […]
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.
Living With Lysosomal Disease
In a break with the past, Einstein’s 2015 celebration of Rare Disease Day focused not on the idea of rare disease generally but instead highlighted the lives of individuals living with a distinct group of 60 syndromes known as lysosomal storage disorders (LSDs). This focus was chosen as a way of celebrating […]
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 disease […]