dandy-walker.org
Dandy-Walker Alliance » CureTheProcess
http://dandy-walker.org/curetheprocess
The Dandy-Walker Alliance is a partner in this campaign. Please learn how you can help. Strives to inspire science-driven public policy that will increase the predictability of the regulatory process for rare disease treatments. Our goal is to give even the rarest diseases access to the accelerated approval process and fulfill more completely the original intentions of the Orphan Drug Act. The following goals need to be reached to put orphan treatments on the fast track:. Due to the rarity of the disorde...
teamsanfilippo.blogspot.com
Team Sanfilippo: The Sad Truth About the US Rare Disease Drug Approval Process
http://teamsanfilippo.blogspot.com/2011/02/sad-truth-about-us-rare-disease-drug.html
Tuesday, February 22, 2011. The Sad Truth About the US Rare Disease Drug Approval Process. When we were first found out that our son had elevated urinary GAG levels (a marker for MPS diseases) we assumed he had Hunter Syndrome. Without treatment, Hunter Syndrome is - like all MPS diseases - an absolutely catastrophic disease. When I went to the Shire website to research treatments, I saw the following quote regarding the efficicacy of Elaprase:. Since no treatment is approved, Sanfilippo parents are extr...
teamsanfilippo.blogspot.com
Team Sanfilippo: February 2011
http://teamsanfilippo.blogspot.com/2011_02_01_archive.html
Sunday, February 27, 2011. The Inequity of Rare Disease Knows No Boundary. That is why on International Rare Disease Day, I want to support the efforts of my good friend Simon Ibell. Please take a moment to check out what Simon is doing and help us spread the message:. HEY CANADA, ON FEB. 28TH, BE FAIR TO RARE. Links to this post. Tuesday, February 22, 2011. The Sad Truth About the US Rare Disease Drug Approval Process. In a clinical study of 96 patients with Hunter syndrome, weekly treatment with ELAPRA...
everylifefoundation.org
Faster Track Newsletter: May | EveryLife Foundation for Rare Diseases
http://everylifefoundation.org/faster-track-newsletter-may
PROGRAMS, INITIATIVES AND COALITIONS. Faster Track: August 2016. Faster Track: July 2016. Faster Track: June 2016. Rare Disease Scientific Workshop. Rare Disease Week on Capitol Hill. Italian Street Painting Marin. August 16th, 2016. FasterCures Wants Your Rx for Innovation read more. August 9th, 2016. NorthBay Biz Profiles Foundation President and Founder Emil Kakkis as a GameChanger read more. August 4th, 2016. Enter your contact information to receive updates and/or invitations from EveryLife. Is pass...
georgiapku.org
Georgia PKU Connect :: News & Events
http://www.georgiapku.org/NewsEvents.html
News About Georgia PKU Connect. Will be held August 26 - 28 in Winder, Georgia! For registration and volunteer information, please contact Camp Director Adele Hunt at. If you or your child has a genetic metabolic condition, you don’t have to walk this journey alone. Get connected and feel supported! Fill out our Get Connected Form. Georgia PKU Connect donates $10,000. More news about Georgia PKU Connect. Georgia PKU Connect Events. August 26 28, 2016. Registration closes August 11th! Donate to the Cause.
taylorstale.org
Rare Disease Links
http://taylorstale.org/rare-disease-partners/links
Taylor’s Tale: One Little Girl’s Story. Other Treatment Approaches for Batten Disease. Visit the sites below to learn more about Taylor’s Tale’s partners, rare disease organizations, and to meet other affected children who, like Taylor, who are fighting for their lives.*. Batten Disease Support and Research Association (BDSRA). Beyond Batten Disease Foundation. National Institutes of Health (NIH). National Institute of Neurological Disorders and Stroke (NINDS). Rare Disease Legislative Advocates.
psp.org
Organizational Resources | CurePSP
http://www.psp.org/education/resources
Patient and Care Partner SUPPORT. Cultivating Patience as a Caregiver. Patient Engagement Program (PEP). In Memory of Gretchen, The Pacific Crest Trail. In Honor of Andrew Szczerba and Supporting PSP Research. Sykes and Cooper Farms Corn Maze. Wine in the Hill Country: in Honor of Mark Witt. Sheree Jacobs For The Cure. OCT 28, 2016 International Research Symposium. OCT 28, 2016 International Research Symposium Registration. Women's Lunch, June 14th, 2016. Fundraising Activities and Ideas. National Center...
bdsra.org
Batten Disease Advocacy and Awareness - Batten Disease Support & Research Association
http://bdsra.org/bdsra-advocacy
Batten Disease Support and Research Association. What Is Batten Disease? What Is Batten Disease? Locate a Testing Center. Family and Caregiver Support. Nuts and Bolts of Care. BDSRA Centers of Excellence. Research and Clinical News. Learn About Clinical Trials. Our Staff and Board. You are here: Home. Batten Disease Advocacy and Awareness. Batten Disease Advocacy and Awareness. In 2013, Batten disease advocates appeared on program panels of the National Institutes of Health and National Institute of Medi...
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