by CheckRare Staff | Oct 16, 2023
Elijah Stacy, 22-year-old author of “A Small If”, founder of Destroy Duchenne, and Capricor Therapeutics Consultant gives his advice to patients and families dealing with Duchenne muscular dystrophy. Transcription: I wrote my book, “A Small...
by CheckRare Staff | Oct 12, 2023
Ciliopathies are a group of rare inherited genetic disorders and have long posed a significant challenge to the medical community. These diseases, affecting the function of cilia, microscopic finger-like organelles found in most cells, have devastating consequences...
by CheckRare Staff | Oct 9, 2023
Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement for EveryLife Foundation for Rare Diseases, describes a new study showing the economic issues correlated with a delayed diagnosis of a rare disease. Transcription: Hi. I’m Annie Kennedy. I’m...
by CheckRare Staff | Oct 8, 2023
Gustavo A. Heresi, MD, MS, Director of the Pulmonary Vascular and Chronic Thromboembolic Pulmonary Hypertension Program in the Department of Pulmonary and Critical Care Medicine in the Respiratory Institute at Cleveland Clinic discusses the challenges of...
by CheckRare Staff | Oct 4, 2023
Steve Hughes, MD, chief medical officer at Avidity Biosciences, explains the mechanism of action for AOC 1044 as a Duchenne muscular dystrophy (DMD) treatment. DMD is a rare genetic disorder due to mutations in the Dystrophin gene, which encodes the...
