Does an ugly holiday sweater help you get into the seasonal spirit?
How about one with unicorns!
Better yet, this sweatshirt also helps find a cure for Sanfilippo Syndrome.
So what are you waiting for? Grab what’s sure to be you and your kids’ favorite holiday shirts this year, and enjoy knowing that you’re also helping cure kids with Sanfilippo Syndrome.
The shirt’s artwork features prancing unicorns, evergreen trees, snowflakes, and the reminder that “All I Want For Christmas Is A Cure.”
Shirts are available in adult and children’s sizes. Order soon to ensure delivery in time for the holidays.
Proceed of the 2019 ugly holiday sweater benefit Cure Sanfilippo Foundation.
*Special thanks to the Sarkar family for the idea, design, and modeling by big sister Sophia!
The goal for 2019 Giving Tuesday, Dec. 3, was a big one: $100,000. Ambitious and hopeful, the Foundation collaborated with its partners families on a plan to far surpass the 2018 record of $43,000. At end of Giving Tuesday, more than $130,000 had been raised.
Families of children with Sanfilippo Syndrome and their supporters hosted more than 50 individual fundraisers via Facebook in the week leading up to Giving Tuesday.
Donation gives children with Sanfilippo Syndrome the gift of more good days. And parents more quality time with their terminally-ill child. Plus … it may just save their lives.
A donation helps keep Carson playing baseball.
Keeps Liv laughing and smiling.
Keeps Spencer splashing in the pool.
Keeps Sadie singing her favorite songs.
Keeps Connor playing basketball and soccer.
Keeps Oliver giving hugs to everyone he meets.
Keeps Eliza playing in the backyard.
Keeps Will eating the pie he loves.
Donation helps fund promising research projects that could extend children’s quality of life, giving them and their families more good days together.The Foundation provided content such as graphics, sample posts, videos, and funny takes on trending memes to support the individual fundraisers.
“Once again our Sanfilippo community came out strong. With all 53 Cure Sanfilippo Foundation fundraisers going on for Giving Tuesday, you have helped raise over $120,000!!, said Lisa Bittner, mom of Luke (MPS IIIA), posted on her 2019 Giving Tuesday Facebook fundraiser. “That is an incredible amount, and will make an impact on moving research forward, faster.”
“We are always humbled by the love and support that our family receives not only today but everyday,” said Josephine Shamaly, mom of Ellie (MPS IIIB), on her 2019 Giving Tuesday Facebook fundraiser.
Several supporters waited until 8:00 a.m. ET on Giving Tuesday to compete for Facebook’s $7 million in matching funds. The competition for those matching funds is fierce and it will be weeks until the Foundation knows whether any of those 8:00 a.m. donations were able to snag a match.
Last month, the research institute TIGEM presented preliminary results of two studies funded by Cure Sanfilippo Foundation at the Conference of Telethon Fundamental Associations in Italy.
Cure Sanfilippo partnered with TIGEM (Telethon Institute of Genetics and Medicine) on research projects to explore re-positioning and re-purposing FDA-approved compounds to determine if identify they can activate the clearance of pathologic lysosomal accumulation in MSD cellular models independently of the missing enzyme in each particular disease.
Posters were presented by TIGEM at the conference for both projects. Further work to determine efficacy and proper concentration doses and delivery is ongoing in animal models and efficacy.
The team developed a new high-content screening assay to determine lysosomal activity. They have identified similar primary “hit compound” in MSD and MPS IIIA. The same compound in the screening reduces cellular heparan sulfate GAGs in cell models for MSD and MPS IIIA.
Read more about each specific research project:
Fellow Sanfilippo Advocate Katia Moletta (mother to Francesco, MPS IIIA) was able to be attend the scientific meeting and connect with the presenters from TIGEM.
“This shouldn’t happen! Especially not to children.”That’s what you thought when you first heard about Sanfilippo Syndrome. You put yourself in the shoes of these parents who were told that their children will suffer and die from Sanfilippo, and it broke your heart.
But you made the remarkable decision to something to help.You donated to Cure Sanfilippo Foundation to find a treatment or cure. And your donation is an investment to create a different future.
You can see how your donation is helping cure Sanfilippo Syndrome.
More research is happening than ever before. More clinical trials than ever in the history of Sanfilippo. Including more than 20 projects and multiple clinical trials funded by Cure Sanfilippo in five years. And much more to come.
Get a deeper look at what your generosity has already fueled. See the faces you are helping and the future you are creating.
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Thank you for your generous support. A cure is closer than ever because of you.
Dad, Donor, President
Cure Sanfilippo Foundation
The community of Dobbs Ferry, NY, turned out on a Saturday morning in November for the 2019 Moonball Kickball Tournament, benefiting Jacob Moon, a local child with Sanfilippo Syndrome.
The second annual Moonball again attracted support from the community for a day of family-friendly kickball, food, and other games. In addition to players, the event was supported by sponsors and volunteers who helped set up, picked up food, manned tables, organized teams, donated items, cleaned up. Students from the middle and high schools helped with the event, and the Mercy College Men’s Lacrosse team refereed the games.
One of the most touching moments of the day was high-school students and teachers organizing a special inclusive “Buddy Ball Game” so Jacob and 10 fellow special-needs friends could join in the fun. Each child was accompanied by two students who helped him/her kick the ball and guided him/her in running the bases. Because of Sanfilippo, this wouldn’t be possible for Jacob without the thoughtful inclusion and accommodation of these students.
“This was definitely one of many highlights of the day,” said Bill Moon, Jacob’s dad. “I loved this for the kids who participated and the volunteers!”
The local news station, News 12 – West Chester, featured the the 2019 Moonball Kickball Tournament.
Last year, Moonball raised $12,000, and they were hoping to surpass that this year. They were successful and raised $18,000 this year! A portion is donated to Cure Sanfilippo Foundation to help fund continued research for a treatment or cure for Sanfilippo Syndrome.
Read an article about the 2019 Moonball by local newspaper The Hudson Independent.
Learn more about Jacob and hear his family’s story about living with Sanfilippo Syndrome.
Adam Shaywitz, MD, PhD, has joined the board for Cure Sanfilippo Foundation, bringing additional scientific and treatment development expertise to the organization.
“We are absolutely thrilled to welcome Adam to the board and are grateful for his support of the Sanfilippo community over the years,” said Cure Sanfilippo Board member Valerie Byers, PhD. “His extensive experience in the drug development process for devastating rare pediatric conditions will provide the Foundation with unique and invaluable guidance as we aim to accelerate our impact in the space for children with Sanfilippo Syndrome.”
Adam received his degrees from Harvard Medical School and trained in adult medicine at Massachusetts General Hospital. He subsequently pursued a fellowship in endocrinology, diabetes, and metabolism jointly at the Beth Israel-Deaconess Medical Center and Joslin Diabetes Center where he concurrently pursued postdoctoral studies in molecular physiology.
Adam is Chief Medical Officer (CMO)-in-Residence at BridgeBio Pharma, where he currently serves as CMO for three subsidiary companies: CoA Therapeutics, Aspa Therapeutics, and Adrenas Therapeutics.
Prior to BridgeBio, he spent five years at BioMarin Pharmaceutical as an Executive Director in the Clinical Sciences group. While at BioMarin, Adam developed, designed and led natural history and clinical studies for Sanfilippo Syndrome. Additionally, he played a key role in developing a number of programs aimed at treating a broad range of diseases, including other lysosomal storage disorders. During his tenure at BioMarin, Adam also worked closely with the research and business development teams to develop and broaden the pre-clinical and clinical pipeline.
“I am honored and excited to continue working with the Sanfilippo community in their mission to identify therapies that could make a meaningful impact to patients and families suffering from this devastating disease,” said Adam Shaywitz. “In addition to the urgency and drive that Cure Sanfilippo Foundation brings to their mission, they also possess a sophisticated understanding of what it takes to deliver novel therapies to patients and a willingness to dive in and participate in this process. I am looking forward to being a part of this dynamic and passionate team.”
Last year, Spencer’s Sprinters raised just over $20,000 for Cure Sanfilippo Foundation by running the Genesis Battlegreen Run in Lexington, MA. This year, Spencer’s Sprinters are crushing last year’s fundraising and are on their way to raising $30,000 in advance of the Nov. 3 race day.
LexFUN! and Lexington Neighbors and Newcomers’ Club organize the annual Spencer’s Sprinters effort on behalf of the Smiths, a local family whose son Spencer has Sanfilippo Syndrome.
Like all toddlers, Spencer loves to splash in the pool with his daddy, dance in the yard with his mommy, and pet every puppy dog he can get my hands on. But unlike most boys and girls, he’s battling MPS-III, commonly known as Sanfilippo Syndrome.
Sanfilippo Syndrome is a genetic neurological disorder that can most-easily be understood as “childhood Alzheimer’s.” Both are a death sentence, but where Alzheimer’s strikes people toward the end of their lives, Sanfilippo attacks kids just as they have begun to learn to walk and talk.
There are several teams of researchers around the world who are studying ways to treat Sanfilippo kids like Spencer who lack an enzyme that breaks down natural cellular waste.
The funds raised by Spencer’s Sprinters helps Cure Sanfilippo Foundation architect and fund more research to treat and/or cure this terrible disease, as well as increases awareness of Sanfilippo.
“It is absolutely amazing to see the local community continue to rally around Spencer and his family,” said Glenn O’Neill, President of Cure Sanfilippo Foundation. “Their generous outpouring of love and support for this family means the crucial research for a cure will continue. It also gives hope to every parent of a child with Sanfilippo. We thank Spencer’s Sprinters for their tireless work and wish them a great run on Sunday.”
Visit the official Spencer’s Sprinters fundraising site and see the impressive number of people helping raise funds to help Spencer and other children with Sanfilippo.
Learn more about Spencer and hear his parents’ thoughts about life with Sanfilippo Syndrome.
*** UPDATE – Nov. 4, 2019 ***
Spencer’s Sprinters sprinted past their 2019 goal and crossed the finish line raising $33,330 to benefit Cure Sanfilippo Foundation and research to cure Sanfilippo Syndrome with this year’s Genesis Battlegreen Run.
Cure Sanfilippo Foundation is among 107 organizations that co-signed a letter earlier this month to U.S. Rep. Peters (CA) and Rep. Shimkus (IL), thanking them for introducing HR 4144, the Ending the Diagnostic Odyssey Act.
The legislation would allow states to conduct a three-year pilot program to increase the Federal Medical Assistance Percentage (FMAP) rate to provide Whole Genome Sequencing clinical services for children on Medicaid with a disease that is suspected to have a genetic cause.
Eighty percent of all rare diseases have a genetic cause. And rare disease patients average eight physicians and three misdiagnoses over the span of seven years before the correct diagnosis is discovered. This “diagnostic odyssey” causes patients to miss critical windows of opportunity for treatment, improved quality of life, and clinical trials, in addition to experiencing anguish and chronic uncertainty.
“The diagnostic resource that HR 4144 would create expedites early and accurate diagnoses of rare diseases, ending the emotional roller coaster of the ‘diagnostic odyssey,'”said Cara O’Neill, MD FAAP, Chief Science Officer for Cure Sanfilippo Foundation.
“Earlier diagnosis means patients can immediately start treatment, if available, that sustain or improve their quality of life or access a clinical trial for an experimental therapy,” said O’Neill. “This is especially critical in diseases like Sanfilippo Syndrome, where time is not on the side of patients.”
The letter, spearheaded by Genetics Alliance, is also being delivered to other members of Congress, requesting their co-sponsorship of this important bi-partisan bill. Select Senators are being asked to introduce companion legislation in that chamber.
The first-ever gene therapy clinical trails, testing whether the treatment will work in children, are happening for Sanfilippo Syndrome Type A and B after decades of work by scientists, families, and foundations, including Cure Sanfilippo Foundation.
Enabling a clinical trial to happen for Sanfilippo Syndrome Type C is among the priorities of Cure Sanfilippo Foundation. And families of children around the world with Type C are helping.
Recently, Kampen Mot Klocken (Fight Against the Clock), a Swedish Sanfilippo group, raised $23,000 and donated it to Cure Sanfilippo in honor of Eyra who has Sanfilippo Syndrome Type C.
Kampen Mot Klocken raised the funds through creative and sometimes-frigid events throughout the year.
“These funds were raised THANKS to volunteers who arranged dance shows, running events, a farmers day, and even a Christmas Day dip in the freezing ocean!” said Eyra’s mom . “But most of it comes from many small donations from all over Sweden – people who just wanted to help us. We have so much gratitude to both our amazing volunteers and donors.”
“These funds will go directly toward moving the Type C program forward toward a hopeful gene therapy clinical trial,” said Foundation President Glenn O’Neill. “Cure Sanfilippo is so thankful for this support from around the globe.”
Eyra’s family is one of many Type C families globally working to support the Foundation’s Type C program, including the partner families of Connor, Jonah, Mia, and Saga & Maia.
The children and their families who battle Sanfilippo Syndrome have superhero levels of resilience, strength, and courage.The Superhero Project brings the superhero within children facing serious illness or disability to life through unique art pieces, and has recently captured the superheros that live inside several children with Sanfilippo Syndrome.
Founded in 2016 by Lisa Kollins, The Superhero Project transforms children into superheroes through vibrant posters created by professional artists from around the world. The families are personally interviewed by one of The Superhero Project’s volunteer sidekicks and walked through a series of questions that explore the superhero that lives inside – how they make the world a better place, what they stand for, what tools they need, what they wear, and more. The information is shared with The Project’s professional animators, comic book artists, and graphic designers, who volunteer their time to turn the ideas into reality and create a fully-realized color image of each one-of-a-kind superhero. Each family receives an 11″ X 17″ poster, plus a digital copy. All at no cost to the families.
“People often comment that the children, parents, and siblings facing Sanfilipo Syndrome are ‘superheros,’ so it’s incredibly moving to see that sentiment brought to life. And in a way that’s unique and specific to the personality and passions of each child,” said Glenn O’Neill, President of Cure Sanfilippo Foundation and father of Eliza. “These compassionate artists volunteer their talent and time to create superheros and super stories, which lifts the spirits of children with terminal illnesses and of the parents grasping onto HOPE.”
Recent illustrations bringing to life the inner superhero of children with Sanfilippo Syndrome:
DRAGON MASTER LUKEThis adventurous and outgoing superhero has the best sidekick ever – Toothless! Dragon Master Luke soars through the skies, spreading joy wherever he goes. His special intuition shows him who most needs a loving hand to hold, and his magic shovel helps him scoop up happiness to share. They crayons, markers, and paintbrushes in his tool belt help him create beautiful images that inspire people. Of course, most inspiring is Dragon Master Luke himself and his buddy, Toothless. They have lots in common, from their giant smiles to their AFOs. You can’t miss this dynamic duo – giant silent fireworks (to make sure that even kids with sensitivity to noise aren’t bothered) accompany them along their paths. Inspired by Luke (age 7) and created by Miranda Pavelle.
“It’s so incredibly beautiful, and so much Luke! Luke doesn’t have the biggest attention span, but when I showed him the poster, he just stared at it. He loves Toothless and his shovel so much, and I can only imagine what he thought when he saw himself drawn riding his favorite dragon, holding one of his favorite things.” – Luke’s parents
WILL POWERWith a smile that never quits and a heart that’s never met a stranger, Will Power is the newest superhero on the block! He inspires people to never give up and to be thankful for the blessings we have–big blessings like love and family, and little blessings like the perfect slice of peach pie. He runs with lightening speed wherever he’s needed and doesn’t let a locked door stop him. His magic keys give him the power to enter (or to escape!) any room in the universe. Will Power is happy to meet people, help them and make them smile–the world needs more superheroes with the power to connect people like he does! Inspired by W. (age 9) and created by Sarah Chalek.
“We are so grateful for the The Superhero Project and the artist for immortalizing our in-home hero and capturing his personality and energy so perfectly! Will was so excited to see himself as a hero!!!” Will’s parents
FOREVER EMILYForever Emily carries the power of the whirlwind, the beauty of nature’s branches and the spirit of the stars everywhere she goes. This heart warrior inspires all who meet her with her energy and loving soul – even if she can clear a shelf at Target in seconds, she’s a character you simply must adore. Forever Emily has a collection of water pails that she uses to summon whirlwinds that bring strength to the people around her. She stands for survival; running, climbing and jumping through the night in her sparkly sneakers, free to move and to live in love. Inspired by E. (age 8) and designed by Taryn Cozzy.
The newest superhero in town is sweet and lovable, but don’t be misled – he can destroy a room with his amazing energy in seconds! Super-J is here to remind the world how important friendship is. Protected by his shield and his big moon heart, he’s armed with everything people need to have fun – Uno, friendship bracelets, Goldfish crackers, and more. He flies from place to place with smoke trailing behind. (No flames – safety first!) With the confidence of Buzz Lightyear, the wackiness of Stitch, and a smile that lights up a room, Super-J is ready to grab your hand, make friends, and take you to infinity and beyond! Inspired by J (age 9) and created by his brother and sister. Designed by Eric Angel Pabon.
ELIZA THE INSPIRATIONALPerseverance and strength – that’s what this unique superhero so perfectly exemplifies. Eliza the Inspirational is as powerful and joyful as an Olympic swimmer, with energy to spare. Wearing her striped socks and water shoes, she relies on super-speed running to carry her across the ocean waves she commands with a flick of her wrist. Eliza dares to dream of what seems impossible: a world where every child is happy, healthy, strong and free. Perhaps she knows more than we do, since she sees the future with her magical headband, its aquamarine jewel glowing ethereally. She carries a satchel filled with water books that inspire people with their stories of strength and courage – her beautiful eyes and big smile inspire them too! Inspired by E. (age 9) and designed by Sarah Wilson.
HENRY, THE HAPPY HAMMERWhen life gives him lemons, this amazing superhero makes lemonade! Henry – The Happy Hammer zooms from place to place with his canine sidekicks Bailey and Carl hitching a ride. With his courage and strength, he protects his family from foes large and smile, and reminds them to look for the bright side of life. He has an amazing ability to be happy in almost every situation! With a smile as bright as the twinkly stars on his shirt and in the skies, Henry – The Happy Hammer will laugh his way right into your heart. Created by H. (age 5) and designed by Jen Davreux.