Sanfilippo Syndrome Advocacy Hub | UniteRare

At a Glance

Live from database

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2Recruiting trials

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3Specialists

Regulatory Watchboard

3 events

Mar 2026Study of Cannabidiol in Sanfilippo Syndrome

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center — PHASE2, PHASE3

TrialNOT YET RECRUITING

Dec 2024An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III

Ozlem Goker-Alpan — PHASE2

TrialRECRUITING

Apr 2016Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Ultragenyx Pharmaceutical Inc — PHASE2, PHASE3

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Sanfilippo Syndrome.
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Clinical Trial Landscape

2 active trials

1Phase 2

1PHASE2, PHASE3

2Total recruiting

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Recent News & Research

No recent news articles indexed yet for Sanfilippo Syndrome.
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Specialist Network

Top 3 by expertise

Ozlem Goker-Alpan, MD

Lysosomal & Rare Disorders Research & Treatment Center, Inc.

📍 Fairfax, Virginia

Score 89 trials

Nathalie Guffon, MD

Hospices Civils de Lyon

📍 Dallas, Texas

Score 31 trial

SungYoon Cho

Samsung Medical Center

Score 01 trial

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Financial Assistance

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