On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler June 10. My stem cell coordinator…
According to a story from BioSpace, the biopharmaceutical company Mereo BioPharma Group recently announced that its investigational therapy setrusumab has earned Rare Pediatric Disease designation from the US Food and…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
Seelos Therapeutics and Duke University have both signed a Sponsored Research Agreement, meaning they will work in tandem to evaluate and test SLS-004, a Parkinson's disease treatment. Their research aims…
Healio recently published interviews with members of UCLA’s DRC Health Training and Research Program conducted by Infectious Disease News. Only a few months ago, WHO had announced the end of…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
CTI BioPharma has just announced that they are submitting a NDA for Pacritinib as a new option for myelofibrosis patients who are facing severe thrombocytopenia. The thrombocytopenia is a result…
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