PHOENIX NEST BIOTECH

Help Advance Clinical Assessments for Sanfilippo Syndrome: Participate in Our Survey ​

To access the Survey, please click here. https://www.surveymonkey.com/r/G7DR59V

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Calling All Caregivers, Physicians, and Educational Therapists

​We invite caregivers (parents and close family members), physicians, and educational therapists who work with and provide care for children with Sanfilippo syndrome types A, B, C, and D to participate in our important survey. Your insights and experiences are vital to our mission of improving clinical outcome assessments for this rare condition.

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What Is the Survey About?

Our survey is designed to gather your unique perspectives on the clinical and behavioral symptoms exhibited by children with Sanfilippo syndrome. We ask that you describe (in detail) symptoms in children with Sanfilippo syndrome. 

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How Will Your Responses Be Used?

Your detailed descriptions will be analyzed by our team, led by professional clinical outcome assessment developer Dr. Paul Williams, PsyD, alongside advanced AI software. These insights will aid in the development of a new clinical outcome assessment tool, which will evaluate symptoms and behaviors observed in video clips of children with Sanfilippo syndrome as they perform activities of daily living. These video clips are being collected through a HIPAA and GDPR compliant video application called C-RARE as part of a natural history study focused on Sanfilippo syndrome types C and D.

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Survey Details & Guidance

• No specialized medical terminology required: Please describe symptoms as you perceive them, drawing    from your personal or professional experience. 

• Don’t worry if you’re unsure about a particular item: It’s perfectly fine to respond with “not observed”

• Take your time:  If needed, you may pause and return to it later. 

• Survey completion deadline: EXTENDED to April 1st, 2026

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We deeply appreciate your time and support!  For additional information you may contact us at caregiver@phoenixnestbiotech.com- Phoenix Nest