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JLK-447 

Is a multi-center, retrospective and prospective observational study of patients with Sanfilippo syndrome type C.  The study will include 3 clinic visits (baseline, 12 and 24-months) where a myriad of clinical, behavioral, and cognitive assessments will be performed. 

 

Additionally, on and between clinical visits prospective real-world data will be collected 6 times at home via C-RARE a video recording application.

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JLK-447 has two sites: 

• Hospices Civils de Lyon - CHU de Lyon, France 

• UTSouthwestern, Texas

For study details and contact information please refer to NCT# 05825131.

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JLK-447 funding support

Funding for this study has come through multiple sources.

• Vaincre les Maladies Lysosomales - Rare mais pas seul: $541,000.00

• Sanfilippo Sud: $120,000

• NIH/NINDS 1R44NS137892-01: 07/01/24-07/01/27 Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidoses IIIC Patients $2,994,701