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Expert Interview

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

Looking at Spruce Biosciences’ Long-Term Data on Tralesinidase Alfa (TA-ERT) in Sanfilippo Syndrome Type B

Ticker(s): SPRB

Who's the expert?

Institution: University Medical Center Hamburg-Eppendorf (UKE) and International Center for Lysosomal Disorders / European Study Group on Lysosomal Diseases (ESGLD)

  • Head of the Lysosomal Disease Unit and Principal Investigator at the International Center for Lysosomal Disorders at UKE and Working Group Leader of the European Study Group on Lysosomal Diseases (ESGLD)
  • Manages dozens of patients with San Fillipino Syndrome and other lysosomal diseases
  • Senior physician and medical specialist in Pediatrics and Youth Medicine with a clinical focus on rare metabolic and lysosomal storage disorders, including mucopolysaccharidoses, Sanfilippo syndrome, and other inherited metabolic conditions

Interview Questions
Q1.

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

Added By: lilly_admin
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?

Added By: lilly_admin
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?

Added By: user1a9v3vo5

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