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Living with Sanfilippo syndrome type A

Stories from people navigating Sanfilippo syndrome type A — diagnosis, treatment, caregiving, and the ordinary days in between.

2 stories

A pediatric developmental assessment kit beside a clinical chart labeled MPS IIIA and gene therapy review.

Neurology

FAYUVI Eligibility Turns on Preserved Function in MPS IIIA

The FDA-labeled population for FAYUVI is pediatric patients with MPS IIIA and preserved neurodevelopmental function, making timely developmental assessment central to treatment selection.

Rayan Salih · October 29, 2026 · 6 min

A pediatric developmental assessment kit beside genetic testing materials in a quiet neurology clinic.

Neurology

Sanfilippo Gene Therapy Hinges on Preserved Function

The first gene therapy for pediatric Sanfilippo syndrome type A is intended for children whose neurodevelopmental function remains preserved, making early assessment and referral central to eligibility.

Rayan Salih · 7 min read

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