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...

How Can Doctors Educate Others About Rare Diseases

  Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement for EveryLife Foundation for Rare Diseases, gives advice to help doctors understand the cost benefits of diagnosing a rare disease early.       Transcription: One of the things that...

How are Acromegaly Patients Managed?

Monica Gadelha, PhD, Professor of Medicine at the University of Rio De Janeiro discusses symptoms and how symptoms for Acromegaly patients are managed.       Transcription: There are two main problems with managing acromegaly. First, let me define...

Advice For Telling Your Story

Elijah Stacy, author of “A Small If”, founder of Destroy Duchenne, and Capricor Therapeutics Consultant says…” , says “You can’t hold anything back if you’re going to tell a story that’s supposed to help people overcome their suffering”.   ...