Happy Thursday, Friends! It's getting cold outside! This week we're highlighting a new patient story from the Czech Republic on a younger patient diagnosed with a disease generally associated with…
According to a story from pharmanewsdaily.com, the gene therapy company Avrobio has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AVR-RD-02, an…
According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…
According to a story from BioPortfolio, the biopharmaceutical company Collaborative Medicinal Development, LLC has recently announced that the first patient has been enrolled in a clinical trial that will test…
Happy Thursday everyone! This week, we're highlights two stories about patients taking action to solve problems in their life: one patient innovates to improve their care, and another starts a…
According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…
A recent study, published in Nature, examined whether or not stem cells could be modified to reduce their chance of rejection. Stem cell therapies are utilized in a wide array…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
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