According to a recent article, researchers in Europe have created a fluorescent biosensor to look at the signaling molecule orexin in mice brains to help understand narcolepsy. Narcolepsy Narcolepsy and…
In a news release from early February 2022, clinical-stage biotechnology company Eureka Therapeutics, Inc. (“Eureka”) shared that two of its therapies – ET140203 and ECT204 – received Orphan Drug designation…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In late January 2022, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") shared via news release that the first patient had been dosed in the Phase 1/2 ADventure clinical trial. Within this…
The FDA has just announced that they have granted Orphan Drug Designation to an investigative treatment called eplontersen. This therapy is being studied as a treatment option for transthyretin (ATTR)…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
48 years of marriage, 2 children, 2 nephews, 7 grandchildren, and 3 great-nieces. It is clear that Lorrie and Stewart Altman, over their time together, have created a beautiful life.…
Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…
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