News and information on issues relevant to you as a parent, caregiver, and advocate of a child with Sanfilippo Syndrome.
News From the Foundation
Connor Beats Sanfilippo online campaign crosses $150,000 for Type C
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...
Foundation Update | New Project & Progress – May 2020
This spring hasn’t been easy for anyone. But many generous people have made it a little less stressful for families of children with Sanfilippo Syndrome (MPS III). People's support of and generosity to Cure Sanfilippo Foundation have been the rainbow in a storm of...
Cure Sanfilippo funds $380,000 for first MPS IIIC gene therapy
Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS IIIC Gene Therapy COLUMBIA, SC (May 15, 2020) — Cure Sanfilippo Foundation is thrilled to announce funding of $380,000 to the New York-based biotechnology company Phoenix...
Devoted mom shaves her head for Cure Sanfilippo, raise awareness
On May 10, 2020 ... Mother's Day in the United States ... Jill Wood shaved off her lovely red hair to raise awareness and fundraise for research to cure children with Sanfilippo Syndrome, like her 13-year-old son Jonah....
“Light Up the Night” on May 15 to honor MPS Awareness Day
"LIGHT UP THE NIGHT" for MPS on Friday, May 15. Friday is a big day in the MPS community every year. It is International MPS Awareness Day. This Friday, May 15, when it gets dark, families, friends, and supporters will light candles outside their homes -- in their...
Inspiring generosity on GivingTuesdayNow; $146,202 in donations
Uplifting Support Seen on GivingTuesdayNow Supporters around the world answered the call of "A Cure Can't Wait" and donated an astonishing $146,202 on GivingTuesdayNow, May 5, to Cure Sanfilippo Foundation. Thirty-three families of children with Sanfilippo Syndrome...
Combined response to FDA draft guidance on drug development
On May 5, Cure Sanfilippo Foundation submitted the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome). The 39-page...
Sanfilippo caregivers invited to take RDCRN coronavirus survey
The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. The Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH), is conducting a...
A Cure Can’t Wait – GivingTuesdayNow
Life looks quite unfamiliar for all of us. But we take heart because one thing hasn’t changed: people are still thinking of others, finding ways to make a difference. Which is why families with Sanfilippo Syndrome are asking for help. Right now. Like everything else,...
Survey: Caring For A Child With Rare Genetic Disorder During COVID-19
Families with a rare genetic disorder such as Sanfilippo Syndrome will have a different experience than others during the COVID-19 emergency. Scientists at UCLA are working directly with families to better understand specific challenges and when they occur by...
TIGEM identifies CLR01 as new potent drug candidate for MPS IIIA
Dr. Fraldi and his team at TIGEM (Telethon Institute of Genetics and Medicine) in Italy have been working to identify a new treatment strategy for neurodegenerative lysosomal storage diseases. Cure Sanfilippo Foundation has supported this study thanks to the support...
Foundation partner RDMD secures $14 million for expanded work
RDMD, which has partnered with Cure Sanfilippo Foundation and other select patient advocacy groups, announced it has raised $14 million in Series A financing to continue its work to accelerate rare disease research and drug development. RDMD has developed an FDA-ready...
What does “better” mean and who gets to decide that?
Dr. Cara O'Neill, Chief Science Officer for Cure Sanfilippo Foundation, posed this question regarding study endpoints to an audience of before the audience of research scientists, biotech leads and industry partners, physicians, U.S. Food & Drug Administration...
Coronavirus & Sanfilippo Syndrome: What Every Parent Should Know
How to Navigate Coronavirus As A Caregiver For A Child With Sanfilippo Syndrome The Coronavirus pandemic adds another layer of complexity and concern to people and families dealing with chronic illnesses and diseases such as Sanfilippo Syndrome. The concerns of...
“George to the Rescue” show renovates Sanfilippo family’s home
"5-year-old Cameron Hyman suffers from Sanfilippo syndrome, a rare genetic condition. Six days after the doctors confirm her condition, Cameron's mom Christina was diagnosed with breast cancer. This fighting family is surprised with a living room renovation they truly...
Caregiver Preference Study for Sanfilippo presented to FDA
The urgent mission to save children from the fatal and rapidly-degenerative disease Sanfilippo Syndrome must continue because the disease's devastating effects never stop, no matter what else is happening in the world. With this mindset, Cure Sanfilippo Foundation and...
Information on Coronavirus & people with chronic medical conditions
Parents of children with Sanfilippo Syndrome are already managing a complex medical condition, which can be further complicated by illnesses. The Center for Disease Control has compiled a list of resources about COVID-19 for people managing their own or others'...
Video: Foundation provides scientific update, Coronavirus impact
Finding treatments and a cure for Sanfilippo Syndrome is the mission of the Foundation, it partner families, and families of children with Sanfilippo around the world. The Foundation is ardently continuing this mission on all fronts, including the science/research,...
March 31: NORD Webinar on COVID-19 and Families w/ Special Needs
Are you a rare patient or caregiver with concerns related to the COVID-19 outbreak? Do you need strategies for coping, staying healthy, and reducing anxiety during this uncertain period? NORD (National Organization for Rare Disorders, Inc.) is hosting a special...
How to keep kids occupied and learning while home | Resource List
Kids are home. Now what to do with them?How to keep them occupied, learning, and physically active is on the minds of parents everywhere as we all practice social distancing. Maybe that’s you or someone you know.We want to help.You’ve done so much for the families of...