Establishing Patient-Centered Outcomes for MCT8 Deficiency: Stakeholder Engagement and Systematic Literature Review.

Nina-Maria Wilpert Davide Tonduti Ylenia Vaia Heiko Krude Catherine Sarret Markus Schuelke

Published in: Neuropsychiatric disease and treatment (2023)

We recommend that future trials should define a relevant improvement in "development" and/or other patient-relevant outcomes compared to natural history as treatment goals.

Keyphrases

case report
replacement therapy
public health
combination therapy
skeletal muscle
glycemic control
insulin resistance
smoking cessation