Is Newborn Screening Cost Effective?

Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement for EveryLife Foundation for Rare Diseases, explains how new technologies in newborn screening aren’t being taken advantage of due to their costs.       Transcription: The Everylife...

Use of Paltusotine in Patients with Acromegaly

Monica Gadelha, PhD, Professor of Medicine at the University of Rio De Janeiro discusses the use of paltusotine in patients with acromegaly.       Transcription: GH is secreted, controlled by somatostatin. Somatostatin is a molecule that produces the...

Patient Story: Duchenne Muscular Dystrophy

Elijah Stacy, 22 year old author, founder of Destroy Duchenne and Capricor Therapeutics Consultant, recounts how Duchenne muscular dystrophy affected his childhood.      Transcription: I’m Elijah Stacy. I’m 22 years old. I’m an author....

The Congenital DM1 Rating Scale

Mike Snape, PhD, chief scientific officer at AMO Pharma explains the Congenital DM1 Rating Scale to assess outcome measures in patients with congenital myotonic dystrophy type 1.      Transcription: The Congenital DM1 rating scale is a variant of a scale...