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The Launch of MISSION: HIDE AND HELP

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 […]

By | September 19th, 2017|Uncategorized|Comments Off on The Launch of MISSION: HIDE AND HELP

MPS awareness day is coming, here is an update to cheer us all up.

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 […]

By | April 11th, 2017|Uncategorized|Comments Off on MPS awareness day is coming, here is an update to cheer us all up.

Jill Wood, Rare Disease Congressional Caucus Briefing

 

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 […]

By | March 8th, 2017|Uncategorized|Comments Off on Jill Wood, Rare Disease Congressional Caucus Briefing

Anonymous Donor to Make a $20,000 Donation!

Tomorrow is Our Chance

images-1I 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.

 

Please give what you can tomorrow, and urge […]

By | November 28th, 2016|Uncategorized|Comments Off on Anonymous Donor to Make a $20,000 Donation!

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

ConnectMPS is a patient registry, MPS and ML organizations are working together from […]

By | August 10th, 2016|Uncategorized|Comments Off on The National MPS Society, Patient Crossroads,  & Jonah’s Just Begun Presents: The ConnectMPS Registry

Heroes Among Us-Official Movie Trailer

Screen Shot 2016-07-07 at 4.02.29 PM Click the video to play trailer.

 

Don’t miss this summers greatest event, ‘Heroes Among Us’. Coming July 23rd, 2016. Tickets on sale now: http://bit.ly/JJBHeroes

By | July 7th, 2016|Uncategorized|Comments Off on Heroes Among Us-Official Movie Trailer

BBB for JJB

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

katie2Enjoy all that this event has to […]

By | April 26th, 2016|Uncategorized|Comments Off on BBB for JJB

Why neglected tropical disease and rare disease researchers need each other

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 […]

By | February 25th, 2016|Uncategorized|Comments Off on Why neglected tropical disease and rare disease researchers need each other

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 […]

By | December 30th, 2015|Uncategorized|Comments Off on Why Rare Disease Companies Need the Pediatric Rare Disease Priority Review Voucher

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 | November 11th, 2015|Uncategorized|Comments Off on This Will Be Our Year, We Have Only Just Begun