Scientific News

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...

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,...

Statement on Coronavirus

The world is grinding to a halt because of Coronavirus. Except for one thing that continues at the same pace ... time. And time is the greatest enemy to children with Sanfilippo Syndrome. Coronavirus is impacting the Sanfilippo community on a few crucial and...