by CheckRare Staff | Oct 30, 2023
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...
by CheckRare Staff | Oct 26, 2023
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...
by CheckRare Staff | Oct 24, 2023
Mike Snape, PhD, chief scientific officer at AMO Pharma talks about his journey to studying myotonic dystrophy. Transcription: My name is Mike Snape. I am a PhD Neuroscientist by training. I started my career with an interest in the health conditions of older...
by CheckRare Staff | Oct 23, 2023
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...
by CheckRare Staff | Oct 19, 2023
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”. ...