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Patty Taormino's Fundraiser:

Real Hope for Sanfilippo Syndrome Child at age 22 years!

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BENEFITING: Tsf Incorporated

EVENT DATE: May 28, 2017

Patty Taormino

THE STORY:

Finally a treatment that my son will qualify for! After a diagnosis of Sanfilippo when my son was 6 years old, we were told to enjoy him while he could have fun. His life would end between 10-12 years most likely, as Sanfilippo is terminal usually between the first and second decade of life. Jesse has 2 severe mutations of the disease, which made it reasonable his life would be shorter. We decided at that time to do all we could to help him be the best he could be for however God blessed us with him. I researched and studied endlessly and prayed fervently. I was elected to the Board of Team Sanfilippo Foundation. Over time I became Vice President. We have diligently researched and funded every possibility under the sun of different things to help our kids and potentially cure. The target treatment for years had been gene therapy. After nearly 20 years of research there are now a 1/2 dozen kids in trials as of this yea. It will take years to become available to the general population, and most likely only to the youngest of kids who are not showing mental regression yet. This means 4 or 5 years old and under will only be eligible. This totally excluded my Jesse despite the years of fundraising that have been done by mostly families who now have older kids or kids have passed away. Jesse is now 22 years old, and still has life in him. We have been truly blessed. On May 24, 2017, Team Sanfilippo has partnered with ArmaGen and met with the FDA to bring the latest enzyme replacement therapy (ERT) to all ages of Sanfilippo Syndrome. This ERT is different than ones that have preceded it. It is being trialed in 2 other MPS diseases and proving to cross the Blood Brain Barrier. In the trials of MPS I they are seeing cognitive improvement. This is very promising for the Sanfilippo community and especially my Jesse Jesse is a very probable candidate for this trial and JHH is a site we can do the treatment. Wehave spoke with genetics doc and see no reason why not. We feel very blessed that Jesse is still here, as many have passed a horrific painful death of seizures, involuntary movements, dystonia, intolerance of feeds, organ failure, and more. Too often death is not as peaceful as one would hope. Please help us to fund this therapy for Jesse. A years worth of trial will cost at least 100k possibly more. I personally have looked at many trials and possibilities over the years, and most I would not try at this stage in Jesse's life, but this one I have been watching for 18 months and it can't get here soon enough! I am very excited about the partnership with Armagen and look forward to getting a treatment for my son! Our goal is have treatment starting by the end of 2017 or before. The Hope is now longer a dream, the dream of a better life is finally here, it's just dollars that stands between my Jesse and the trial! Please share and help make our dream a reality! God did not bless us with Jesse for 22 years for nothing! He is meant for this! You can view more about Sanfilippo and the trial at http://www.teamsanfilippo.org


To see more about this ERT and what it's doing in another MPS disease right now, ciick here. https://vimeo.com/217080163

$50

MONEY RAISED
  • Connie Christopher/Chanda Smith

    $50

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0% Raised of$100,000 Goal

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The Team: $50 TOTAL RAISED SO FAR

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Donor Comments

Connie Christopher/Chanda Smith

Connie Christopher/Chanda Smith

DONATION: $50

In honor of my daughter, Chanda, too far into the fight. 2 years ago