Biomarin is full speed ahead with their clinical trial. If you haven’t updated your surveys on our patient registries, now is a good time to do so. Help BioMarin help us. https://connect.patientcrossroads.org/?org=ConnectMPS If you haven’t registered your MPS, ML or Glycoprotein family members, this would be a great way to celebrate MPS […]
Jill was asked to speak at the Rare Disease Congressional Caucus briefing this past week when we were in Washington, D.C. for Rare Disease Week. Although Jill addressed the caucus 2 years ago, she was still a bit hesitant to accept. When she found out she was […]
Tomorrow is Our Chance
I KNOW! We are excited too. If we can raise $10,000 tomorrow, #GivingTuesday, we will get a DOUBLE MATCHING donation of $20,000. On top of that, the charity that raises the most will get a $25,000 donation from Crowdrise.
The National MPS Society, Patient Crossroads, & Jonah’s Just Begun Presents: The ConnectMPS Registry
Join our webinar for ALL STAKEHOLDERS!
August 16, 2016 at 12pm EDT
The online meeting will take place via webinar. To join please visit: http://join.me/386-537-239
TOGETHER WE CAN BRIDGE THE GAP
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 […]