PHOENIX NEST BIOTECH

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

To access the survey link, 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) how you would observe and characterize certain symptoms in children with Sanfilippo syndrome. We anticipate that the responses from caregivers, clinicians, and therapists will reveal both similarities and differences, helping us gain a fuller understanding of the syndrome’s presentation and progression.

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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 specialized video application 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. Your natural observations are highly valued.

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

• Take your time: The survey is comprehensive. If needed, you may pause and return to it later. We want you to feel comfortable and unrushed.

• Survey completion deadline: December 1st, 2025

 

Get Involved & Contact Paul!

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

If you have any additional questions please contact Dr. Williams at P.Williams@phoenixnestbiotech.com.

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Your participation is a crucial contribution to advancing clinical research for Sanfilippo syndrome. By sharing your perspective, you will help create a better tool for understanding and evaluating the symptoms and behaviors that children experience.

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We deeply appreciate your time and support—thank you for helping us move closer to improved care and assessment for children with Sanfilippo syndrome!