ADVANCE 2024: Introduction keynote

ADVANCE 2024: Deep Dive Gene Therapy Considerations

ADVANCE 2024: Impact of Sanfilippo on the Retina

ADVANCE 2024: Deep Dive: Supporting Health Through Nutrition

ADVANCE 2024: The Advantage of Qualitative Interviews in Clinical Trials

ADVANCE 2024: Updates on Clinical Trials for Sanfilippo Syndrome (Part 1)

ADVANCE 2024: Cure Sanfilippo Foundation Programs Affecting Change

ADVANCE 2024: Advances in Preclinical Research For Sanfilippo

ADVANCE 2024: Sanfilippo In My Voice: Experience with Brain & Tissue Donation

ADVANCE 2024: Deep Dive: Mitochondria & Metabolism in Sanfilippo

ADVANCE 2024: Drug Repurposing Studies for Sanfilippo Syndrome

ADVANCE 2024: Evidence-Based Architectural Design Strategies for Enriching Environments

ADVANCE 2024: How does the “Accelerated Approval” process work?

ADVANCE 2024: Updates on Clinical Trials for Sanfilippo Syndrome (Part 2)

ADVANCE 2024: Closing from Cure Sanfilippo founders

ADVANCE 2024: Sanfilippo in My Voice: Our Experience Living with Attenuated Sanfilippo Type B

ADVANCE 2024: In Memoriam Tribute

ADVANCE 2024: "How We Do It" families #1 & #2

ADVANCE 2024: "How We Do It" families #3 & #4

ADVANCE 2024: "How We Do It" family #5

ADVANCE 2024: "How We Do It" family #6

ADVANCE 2024: "How We Do It' family #7

ADVANCE 2024: "How We Do It" family #8

ADVANCE 2024: Closing Remarks at the Regan-Udall Meeting with the FDA

We need a miracle. Save Lydia.

Cara O'Neill | Qualifying Biomarkers to Support Rare Disease Regulatory Pathways | Closing Speech

Life with "Attenuated" Sanfilippo Syndrome | The Kohlmorgan Family

Durable Medical Equipment (DME) and Sanfilippo Syndrome

What is Assistive Technology and how can it help your child communicate?

First time parents watch video they hope will save their daughter