by CheckRare Staff | Nov 8, 2023
DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with DM1[^2^]. Image: Dreamstime This therapy consists of an antigen-binding fragment antibody (Fab) conjugated to an antisense...
by CheckRare Staff | Nov 6, 2023
Joseph Mikhael, MD, Chief Medical Officer at the International Myeloma Foundation explains racial disparities in the diagnosis of Multiple Myeloma Transcription: They don’t have the same incidents level that we see in the African population, but...
by CheckRare Staff | Nov 2, 2023
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...
by CheckRare Staff | Nov 1, 2023
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...
by CheckRare Staff | Oct 31, 2023
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....
