Patient Needs & Culture of the Laboratory; Kickoff 2021 StepUp fundraiser; families milestone & more

A Close Look at Sanfilippo

Episode 5: “A Close Look At Sanfilippo” is a regular 10-minute podcast covering five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no FDA-approved treatment.

In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about a recent presentation by Foundation Chief Science Officer Cara O’Neill, MD, FAAP, who offered multiple recommendations on how the “culture of the laboratory” can better integrate the true needs of patients, a facet of research that is sorely lacking. Additionally, Glenn talked about the kickoff of the 2021 Step Up to Cure Sanfilippo steps challenge and how people are using their steps to help children who have a disease that literally robs them of the ability to walk. He also addresses how newborn screening for Sanfilippo would be a “game changer” and the Foundation’s support of a newborn screening pilot in New York. Glenn also touches on the more than 100 partner families from around the world joining the Foundation and creation of a Professional Advisory Board to allow volunteers to lend their professional expertise to achieving the Foundation’s mission.

#CureSanfilippo #RareDisease #ACureCantWait #ACloseLook #Podcast #NewbornScreening #2021NBS

© 2021 Copyright reserved Cure Sanfilippo Foundation