During clinical trials, researchers work to advance clinical knowledge, build an understanding of certain diseases or conditions, and learn more about a new drug, surgical device, or behavioral modification technology. However,…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) recently hosted a webinar presentation on February 9, 2021. This webinar, titled "Why Clinical Trials Matter and How to Find the Right One for…
Ionis and the University of California, San Diego (UCSD) have formed a team to investigate antisense oligonucleotide technology as a treatment for multiple myeloma. Their drug, ION251, has shown positive…
AVROBIO has released data from the ongoing Phase 2 FAB-GT trial, which is investigating AVR-RD-01 as a treatment for Fabry disease. This ex vivo lentiviral gene therapy was shown to…
According to a story from GlobeNewswire, the biopharmaceutical company Soleno Therapeutics, Inc. recently hosted a Key Opinion Leader (KOL) webinar, which was focused primarily on the latest updates to its…
The United Kingdom has taken a big step in the right direction for rare disease patients, as they have recently created the UK Rare Diseases Framework. This framework is intended…
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…
Hundreds of mutations have been connected to autism spectrum disorder (ASD), and a new study finds further evidence that suggests a possible cause of this spectrum of conditions Neuroscience News…
According to ForexTV, commercial gene therapy company Spark Therapeutics ("Spark") recently announced preliminary data from a Phase 1/2 clinical trial evaluating SPK-8016, an investigational gene therapy, for patients with hemophilia…
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