Tag: Duchenne Muscular Dystrophy

Caregiver risk preferences for delaying loss of ambulation in Duchenne muscular dystrophy

Jason Shafrin

That is the title of a paper published today in Current Medical Research and Opinion with co-authors Nadine Zawadzki, Moises Marin, Ivana Audhya, Lauren Sedita, Natasha Kulkarni and Alexa Klimchak. The abstract is below. ObjectivesQuantify caregiver risk preferences to inform the “value of hope” for Duchenne muscular dystrophy (DMD) therapies affecting time to loss of…

Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy

Jason Shafrin

That is the title of a paper recently published in AJMC with co-authors Suhail Thahir, Alexa Klimchak, Ivana Audhya, Lauren Sedita, and John A. Romley. The abstract is below. Objectives: To quantify the magnitude of altruism value as applied to a hypothetical new treatment for a rare, severe pediatric disease: Duchenne muscular dystrophy (DMD). Study Design: Prospective…