In spring 2019, Connor Dobbyn‘s parents were told their son had Sanfilippo Syndrome, Type C (one of the rarest types), and that it will take his life. Like Alzheimer’s in children, Sanfilippo will cause Connor to lose all the skills he’s gained, stop speaking and walking, suffer seizures and movement disorders, and then die, all in a matter of years. Right before their eyes.
Mike and Marisa decided to fight for their son’s only chance at life: a first-ever gene therapy clinical trial. A clinical trial that needs millions (approximately $5 million for toxicology testing, gene therapy drug production, and clinical trial costs to treat an estimated 10-20) to happen.
Mike and Marisa turned their lives upside down and began fundraising in earnest in August 2019 through multiple channels – social media, in-person events, press coverage, and more.
And on May 29, little more than a year since Mike and Marisa were told Connor has Sanfilippo Syndrome, the online facet of their fundraising crossed the impressive $150,000 mark.
And they’ve only just begun.
Rallied By A Diagnosis
Mike and Marisa made the heart-breaking announcement about Connor’s diagnosis with friends and family on Aug. 23. They shared their desperate hope for a different fate for Connor and their determination to make it happen.
Hundreds of family, friends, co-workers, classmates, neighbors, and more responded to Mike and Marisa’s announcements. The online “Connor Beats Sanfilippo” quickly accumulated more than $30,000. And most importantly, it began to spread around the country and the world because they shared the story with their personal networks.
Donations to Connor Beats Sanfilippo have come from around the world, including Canada, United Kingdom, India, Australia, Singapore, and Belgium.
“I am overwhelmed by the outpouring of unconditional love and support for Connor and our family. There really is strength in numbers and it truly takes a village. I honestly feel blessed that Connor has touched the hearts of so many people who want to come together as a community and fight for his life,” said Connor’s mom Marisa via social media.
The campaign received a huge boost in early 2020 when Connor was featured in the Philadelphia Inquirer in a series of articles by reporter Maria Panaritis.
- “Childhood Alzheimer’s: A Chester County boy and his family are seeking help against a burglar of innocence” on Jan. 25, 2020
- “Inquirer readers donate nearly $20,000 to save Connor Dobbyn from ‘Childhood Alzheimer’s’” on Jan. 30, 2020
- “‘He never gets sad’: Connor has ‘Childhood Alzheimer’s,’ and his little buddies are his rock” on Feb. 23, 2020
Inquirer readers responded passionately and donated roughly $40,000 over the course of six weeks.
Bigger Than Just An Online Campaign
The $150,000 raised online only tells a portion of the fight for Connor. In addition to the online fundraising, there have been dozens of events and fundraisers, hosted by families, friends, neighbors, classmates, and generous strangers.
Even COVID-19 couldn’t indefinitely halt the momentum of Connor’s fight. A local restaurant hosted a carry-out fundraiser at two of its locations in April. More local fundraisers are in the works for the coming weeks.
Progress of the Clinical Trial
The donations benefit Cure Sanfilippo Foundation, a 501(c)3 non-profit (Tax ID 46-432131), that is funding the clinical trial for Type C. Recently, the Foundation funded $380,000 to the New York-based biotechnology company Phoenix Nest, Inc., to support creation of plasmid (a critical component of gene therapy drug production), as well as the required in-vivo efficacy study in the MPS IIIC mouse model. These and other steps will enable the project to be evaluated by the U.S. Food and Drug Administration (FDA) for approval to proceed with a clinical trial.
An Important Moment For Type C
Type C is among the rarest of types of Sanfilippo Syndrome. The more prevalent types of Sanfilippo, Type A and B, have historically received more attention because of their larger patient populations. But Connor’s family and the many others around the world – some of whom have been working towards research specially for Type C for years – are now seeing progress.
Mike and Marisa, along with other families of children with Type C, continue to ardently fundraise to make the first-ever gene therapy clinical trial a reality, and without any promise of treatment for their child. Because they know that without it, there’s no chance at all.