Update on clinical research: Team Sanfilippo is committed to bringing treatments to the patient. We’ve been funding mouse and animal research for over 10 years now and were a major contributor to getting Gene Therapy to the patient. We were the entity that brought Tim Miller, now ABEONA into play here in USA just for the gene therapy trials. We also have been critical in helping two of the major drug companies to establish protocols for ERT for our kids. In that 10 years very little is still available to 95% of the children. We are dedicated to getting these treatments TO OUR CHILDREN DIRECTLY!! They have suffered long enough!
We at Team Sanfilippo have initiated one trial that will be starting very soon. In about 30 days. It is an IV trehalose trial DIRECTLY to 6 children that are moderately affected by Sanfilippo. We are using both type A and B children and have selected families that we know will follow thru with weekly transfusions for this trial. Trial protocol is set up, clinic and doctor are both ready, and as soon as drug production is complete, trial will begin. If this trial proves as successful as we think it will, the trial will open to more children after 6 months.
This trial will cost approx. 60k-70k per child, so we are counting on your donations and fund raising to help pay for these trials.
At the end of May 2017 we will hopefully have the go ahead to start organizing another trial, AGAIN DIRECTLY TO THE CHILDREN. This trial is proven to cross the BBB. This trial would hopefully begin by the end of 2017 or first quarter of 2018. Again we are looking at type B and possibly A side by side with it. We will share more on this after the end of May. Again major funding and donations will be needed, as this trial will be more expensive than the one listed above. Also again we will be treating children and young adults with moderate regression. Not necessarily the newly dx’d . We want to see what the results are ASAP, not years down the road. Treating only the very young just delays the time frame of getting drug to all the kids when we have to wait years to see if there are changes. It also is not a true predictor, as every kid regresses differently and when treating the very young, we have no idea if that child would have regressed at 2-4 years or 8-12 years as some have. So again, many years would go by treating the very young unaffected kids to see if a treatment works. Meanwhile 100’s of children continue to die. It is time to get treatments to ALL our kids ASAP!
Our goal is to STOP Sanfilippo in his tracks and or CURE the disease. We have many things proven to slow the progression that are not being used by many, we want to focus on the fact that slowing the disease gives your children a better chance at being selected for a trial for a cure when they become available. It is very important to prevent and slow damage in the children for the best quality of life. This is what we do best while pushing research to prevent and repair damage in every cell of this disease.
We have been providing product for studies to families such as genistein, CBD oil, sleep monitors,, DNA check ups to see who in the family are carriers. This year it will be doing even more with the two upcoming trials.
Please consider donations, fundraisers, honorariums and partnerships in providing help for all of these children. Thank you for sharing and helping in anyway possible. No amount is too small, it all adds up at the end result.
Again fundraising is all the time, not just on MPS AWARENESS DAY! Thank you and may God Bless all our children!
____________________________________________________________________________________________________
Saturday April 1, 2017 – Lukie’s Legacy Bowling Bash!
REV’s Bar and Grill
275 N. Washburn St. , Oshkosh, ,WI.
Registration 11 am – Bowling 11;30 – 2pm
Lifes’s a Beach is the theme this year, so dress in beach wear. Prizes for best costume and team costumes. There will be raffle prizes and face painting too! Always a fun and exciting event with fun people!
https://www.facebook.com/LukiesLegacy/?fref=ts Lukie’s Legacy link
____________________________________________________________________________________________
Saturday, March 18, 2017 – Colin’s Crew
@Ten Pin Alley
6285 Nesbitt Rd, Fitchburg, WI
(Southwest side of Madison) – This event is great for the whole family. Bowling, Pizza, Raffles nd drawings. Come to bowl, or just come and eat and cheer on your family or friends!
RSVP: Download a Registration Form by 03/03/17 on the event’s
Facebook page: >Colin’s Crew: Bowling For A Cure
(Registration at the bowling alley on the day of the event is possible, if space is
available, but pre-registration will be helpful for planning.)
Contact: Facebook Page: Colin’s Crew: Bowling for a Cure-2017
or e-mail: cureforcolin@gmail.com
2017 03 18 Flier MPS Bowling for a Cure Event < Double click on link for the flier