PHOENIX NEST BIOTECH

ALL-127

All-127, is a single-center, retrospective and prospective observational study of patients with Sanfilippo syndrome type D. Participants assented/consented to participate in two clinic visits (baseline and 12 months) where a multitude of clinical, behavioral, and cognitive assessments were performed.  Additionally, prospective real-world data was collected at home via a recording application, RARE, at baseline, 6, 12, and 18-month time points. NCT # 05648851 

 

This study was completed July 2025.  Phoenix Nest would like to extend a special thanks to the families that participated! We look forward to publishing the study results late 2026.

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ALL-127 has received funding support from:

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NIH/NINDS UB1NS122644: 09/01/2021 - 08/31/2025 Evaluation of clinical outcomes assessment (COA) and potential biomarkers to facilitate Interventional trial for Mucopolysaccharidosis IIID Patients $3,419,934

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