A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole. About the…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
According to a story from offspring.lifehacker.com, medical abnormalities and conditions that affect the skin are often something that many of us will notice on our own. Once a skin problem…
According to a story from Scope, Dr. Megan Mahoney has been spearheading a project with Stanford Medicine that aims to transform the dynamic between patients and their care providers. This…
One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in Dallas, Texas. When: Saturday, November 9th, 2019 Where: Aloft Hotel Dallas Love Field 2333…
A New Collaboration The National Hemophilia Foundation (NHF) is a nonprofit specifically focused on accelerating research for Hemophilia, a rare blood disorder. It was established in 1948 and now has…
According to a story from the 23andMe Blog, Reni Winter-Evans, who was diagnosed with Parkinson's disease, understands that you don't have to be a scientist to be a big part…
Geisinger Health System is based out of Pennsylvania. They have recently initiated a novel program that has goals to make DNA sequencing a standard part of routine clinical care. Next…
According to a story from birminghammail.co.uk, eight month old Archie was diagnosed with a serious rare disorder called Pompe disease. With his life in danger, the infant faces 220 mile…
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