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FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?

FDA Law

Janet Maynard, Director of this Office, highlighted in the press release that “[g]enetic metabolic diseases include very rare diseases that individually affect a limited number of patients. Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”

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Scope This! A Podcast on Gastroesophageal Reflux and Gastritis

PEMBlog

I’ll make the important distinction between gastritis – which is diagnosed only via endoscopy – and dyspepsia, the term best used to describe the symptoms many patients experience. 2013-0421 Tighe M, Afzal NA, Bevan A, et al. So diagnostic testing centers around endoscopy. J Pediatr Gastroenterol Nutr.

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Aging and the ICU: Podcast with Lauren Ferrante and Julien Cobert

GeriPal

This idea that for critically ill patients in the ICU, geriatric conditions like disability, frailty, multimorbidity, and dementia should be viewed through a wider lens of what patients are like before and after the ICU event was transformative for our two guests today. GeriPal podcast with Linda Fried on frailty.