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Children with MPS I around the world are suffering from cognitive impairment and memory loss because of MPS. Dr.'s Agnes Chen and Patricia Dickson at Harbor UCLA's LA BioMedical Research Center in Torrance, California have initiated a study to help treat those who suffer with these horrible signs of MPS. Dr's Chen and DIckson's trial involves placing a small amount of Aldurazyme, the drug the Ryan Foundation provided invaluable funding to help develop (see 60 Minutes link), into the spinal cord where it is then taken to the brain by the patient's cerebral spinal fluid. Sixteen trial participants are being identified from across the US and Canada. Each participant will need to travel to Torrance, California and/or Oakland Children's Hospital in Oakland, California to receive testing and treatment once before their trial begins, once a month for four months, and then once every quarter until the trial concludes. In addition, each participant will need to travel to the University of Minnesota in Minneapolis, Minnesota prior to the trial and twice yearly once the trial begins where Dr. Elsa Shapiro and her team of experts will test and perform detailed MRI's on the participants so that data may be gathered on how the brain is responding to the treatment.

All of this travel is quite expensive. The Ryan Foundation and our partners at UCLA, Oakland Children's, and the University of Minnesota have estimated that $10,000 per month is needed to pay for airfare, hotel, food, and ground transportation for each of the participants and one parent or guardian. That's $10,000 per month for five years. These children deserve the chance to live a healthy life, but they can not achieve their goals without YOUR help. The Ryan Foundation has accepted the challenge to provide all of the necessary funds for these brave and heroic children and adults to travel to receive treatment - the treatment that may provide them the gift they dream of each day... ...the gift of tomorrow.

Every dollar donated to the Ryan Foundation in 2014 will be used to fund the travel for this ground breaking trial. Won't you please partner with others around the world and help these children find their tomorrow's at LA Biomedical at UCLA, Oakland Children's Hospital, and the University of Minnesota? 

You may make donations by using our PayPal account or by contacting us for other payment options. Thank you for supporting our mission of funding science and finding hope for the lives of children in need. 

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E-Book on Rare Disease Research!

Free E-Book: Rare Diseases – Diagnosis, Therapies and Hope

This is a rare diseases E-Book focused on all rare disease audiences and includes:
  • Ten case studies of amazing discoveries from rare disease families
  • Many interviews with top doctors, researchers and genomics experts
  • A Rare Disease Parent’s Toolkit with recommendations from parents and others
  • Demystifying Genomics, which simply explains the latest revolution in medicine with plenty of information on how the sequencing process works.

Visit http://raregenomics.org/e-book. It's free! We are featured in the forward on Page 7, our chapter is found on Pages 49-54, and Dr. Kakkis' chapter, that also has a few pictures of Ryan and Emil’s perspective of things, is on Pages 90-93.