On October 7th, 2021, Patient Worthy took part in RARESummit21, a virtual event organized by the Cambridge Rare Disease Network (CRDN). "...a flagship CRDN event gathering over 300 great minds…
Collaboration can be extremely important when it comes to developing treatments, especially for rare disorders. Sharing information, technology, and expertise can lead to therapies that otherwise might not have seen…
According to a recent news release, the first cohort - consisting of healthy volunteers - was dosed in a Phase 1 clinical trial evaluating an intranasal formulation of 5-MeO-DMT. Ultimately,…
When drug developers receive marketing approval in different countries, they expand medication access and allow patients across the globe to have better treatment. According to Korea Biomedical Review, Korea recently…
A rare disease diagnosis changes your life. As I look back on eight years of twin anemia polycythemia sequence and its impact on my girls, I also have to reflect…
Three doctors recently got together to discuss how they manage care for their sickle cell disease (SCD) patients. As a part of this conversation, they discussed how they combat systemic…
Currently, there are no FDA-approved treatments for nonalcoholic fatty liver disease (NAFLD) or its more advanced version: nonalcoholic steatohepatitis (NASH). This is partly due to the lack of understanding surrounding…
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