Sometimes through tragedy and hard times, a beacon of hope can emerge. After months of struggling with a rare aggressive disease, toddler Chloe Barnes passed away in 2008 in Minnesota.…
According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the…
We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…
One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in conjunction with Kansas University Medical Center, with support from Celgene, Novartis, Takeda,…
According to a story from journals.lww.com, The Lazarex Cancer Foundation and the University of California San Francisco have announced plans to work together on a three year study that will…
According to a story from Biotech 365, the biotechnology company Viela Bio announced the publication of critical results from a recent study that tested the company's investigational drug inebilizumab as…
According to a story from meaction.net, Jaime Seltzer, the Director of Scientific and Medical Outreach at #MEAction, recently spoke at the Third Annual Working Group Meeting on the Molecular Basis…
According to a story from firstworldpharma.com, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the company's experimental product candidate sotatercept has earned Orphan Drug designation from the US Food…
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