Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
As reported recently in SciTech Daily, for years scientists have recognized red blood cells (RBC) as a vehicle to deliver drugs within the human body. They circulate for days and…
As reported in Cystic Fibrosis News; the 2020 Santander Universities Entrepreneurship Awards are part of an initiative to encourage young entrepreneurship via support of student led startups. This year there…
As reported in Newswise, a new experimental study on vaping, a popular electronic device used to consume nicotine or cannabis, has found that the e-cigarette products with nickel-chromium alloy heating…
According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…
According to a recent report in the Journal of Clinical Oncology, over one thousand patients with high-risk stage III melanoma were included in the Keynote-054 clinical trial (NCT02362594). The…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
This week, the US FDA announced that they approved the drug Nucala (mepolizumab) to treat patients with hypereosinophilic syndrome, a rare chronic blood disease which causes organ damage. Nucala is…
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