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Expert Interview

Slingshot members are talking to an expert! The topic is:

A Third Look: Discussing Spruce Biosciences’ Long-Term Data on Tralesinidase Alfa (TA-ERT) in Sanfilippo Syndrome Type B

Ticker(s): SPRB

Who's the expert?

Institution: Cincinnati Children's Hospital Medical Center

  • Professor of Clinical Pediatrics and Director of the Medical Genetics Residency and Fellowship Programam at Cincinnati Children's Hospital Medical Center.
  • Currently manages 2 patients with with Sanfilippo Syndrome Type B (MPS IIIB) and 15 patients with late-onset pompe disease (LOPD) and has experience prescribing Nexviazyme.
  • Research focuses on Fabry disease, Pompe disease, Sanfilippo Syndrome Type B (MPS IIIB), hypophosphatasia, velocardiofacial syndrome, 1p36 deletion syndrome, Neurofibromatosis type I, and other genetic conditions. 

Interview Questions
Q1.

How many patients do you manage with Sanfilippo Syndrome Type B?

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Q2.

On a scale of 1 to 10 (with 10 being ecstatic), how excited are you for Spruce Biosciences’ Tralesinidase Alfa (TA-ERT) as a potential treatment for Sanfilippo Syndrome Type B?

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Q3.

What % of older patients that have progressed might not take this ERT? What prevalence of patients and what % of this disease will you think take this ERT in the US?

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