We are more than excited to launch our new campaign Mission: Hide and Help
This is a grassroots social experiment meant to help diagnose Sanfilippo Syndrome. Aimed at doctors and executed by kids, we are mixing our love of Pokemon with the urgency to find more patients. “Nobody can find rare like you can”.
Read full press release […]
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 […]