Curing Sanfilippo Syndrome is our ultimate goal, but we also want to influence the road to get there. With a 360-degree view, the Foundation funds extensive research into multiple avenues for a treatment/cure, as well as spearheads, supports, and funds initiatives that provide valuable, disease-specific and patient-preference information.
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 LUKE
This 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
With 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 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 INSPIRATIONAL
Perseverance 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 HAMMER
When 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.
As of Oct. 7, Cure Sanfilippo Foundation has again passed the $1 million-raised mark in a year, thanks to the 70+ partner families and the generous donors nationally and globally who are committed to finding a treatment or cure for Sanfilippo Syndrome.
This is the fourth year the Foundation has raised more than million dollars, which is remarkable given that it was founded in late 2013. And there are no plans to slow down in the final three months of the year in order to push towards the annual goal of $2 million.
“The research, projects, and clinical trials that these donations enable the Foundation to fund are helping continue to accelerate medical options for children and HOPE for their families and parents,” said Glenn O’Neill, President and father of Eliza, MPS IIIA. “I’m truly humbled and in awe of the amazing people who donate to help us cure or treat this disease.”
In recent years, the activities and hardwork by families of children with Sanfilippo Syndrome and their friends has created significant momentum. In 2013, there were no clinical trials happening for Sanfilippo Syndrome. Now, there are multiple. More than ever, a cure feels inevitable among the Sanfilippo community. And donations are what will dictate how fast that new future happens.
“Every single donation, no matter its size, is so special,” said O’Neill. “Each one brings us a step closer to saving lives and to giving families better days, and more good days, with their children.”
Three years ago, Glenn and Cara O’Neill took the phone call that would change their family forever.
A call letting them know whether their daughter Eliza would be permitted in a possibly life-saving clinical trial for Sanfilippo Syndrome, a rare, genetic, and fatal neurodegenerative disease likened to a childhood Alzheimer’s.
Thankfully, the news was good; Eliza was accepted into the first-ever gene therapy clinical trial for Sanfilippo Syndrome, Type A, conducted by Abeona Therapeutics and Nationwide Children’s Hospital and followed by a trial for Type B. (NOTE: Both clinical trials are still enrolling patients.)
Months later, Glenn and Cara decided to share that raw and life-changing moment in the hopes of spreading awareness to gain more funding for research for the many Sanfilippo children suffering and in need of a chance at life.
Today, the video continues spreading awareness with people still sharing and viewing it. In April 2019, it surpassed 3 MILLION views on YouTube.
The video also sparked extensive interaction and discussion, with 19,000 likes, 1,800 dislikes, and more than 2,000 comments.
One of the most-common comments was the of question of why the O’Neills chose to record and share the moment. The O’Neills directly responded to the commenters with a thoughtful explanation in the hopes of providing insight to their decision:
“Any time during our journey where we feel a moment is going to be special, we video it so that perhaps we might use it to spread more awareness to gain more funding for research for the so many children suffering and in need of a chance at life. … We will film and post any personal moment if we feel it will help the cause to Cure Sanfilippo and get more funding for clinical trial options to save children.”
“We used to be a private family, but we’ve had to change that to try to get attention for this rare disease. I truly hope no one ever finds themselves in the position we and other families are, and the desperation of having your beautiful child with a rapidly degenerative terminal brain disease with no cure or treatment. Perhaps you understand now why we posted this…even if you don’t agree.”