Sanfilippo syndrome type C

Last reviewed

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ORPHA:79271OMIM:252930E76.2
Who is this for?
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1Active trials1Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Nov 2024Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

Phoenix Nest — NA

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Sanfilippo syndrome type C.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
N/A1 trial
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
N/A
Actively Recruiting
PI: Nathalie Guffon, MD (Hospices Civils de Lyon) · Sites: Dallas, Texas; Bron · Age: 1299 yrs

Specialists

1 foundView all specialists →
NM
Nathalie Guffon, MD
Dallas, Texas
Specialist

Rare Disease Specialist

PI on 1 active trial

Treatment Centers

8 centers
⚗️ Trial Site

Hospices Civils de Lyon

📍 Bron

👤 Jerome Honnorat, Pr

👤 Sebastien HASCOËT

⚗️ Trial Site

The University of Texas Southwestern Medical Center

📍 Dallas, Texas

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

Travel Grants

No travel grants are currently matched to Sanfilippo syndrome type C.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Sanfilippo syndrome type C

Disease timeline:

New trial: Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

Phase NA trial recruiting.

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Sanfilippo syndrome type C

Are there clinical trials for Sanfilippo syndrome type C?

Yes — 1 recruiting clinical trial is currently listed for Sanfilippo syndrome type C on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Sanfilippo syndrome type C?

1 specialists and care centers treating Sanfilippo syndrome type C are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.