by CheckRare Staff | Oct 18, 2023
Mike Snape, PhD, chief scientific officer at AMO Pharma explains the different methods currently being used to treat and quell the effects of myotonic dystrophy. Transcription: Currently, patients with myotonic dystrophy have non-pharmacological support. They...
by CheckRare Staff | Oct 17, 2023
Cheryl Petruk, co-founder of CACHEducation, talks about the need to properly educate patient advocates on the fundamentals of patient advocacy. Transcription Hi, I am Cheryl Patrick from CACHEducation. In the recent past, healthcare has become or...
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...
