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…
Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
124 million cases worldwide, with 2.73 million associated deaths. These are the numbers for COVID-19, a novel coronavirus pandemic which, over the past 12-15 months, has swept the globe. In the…
According to a story from Charcot-Marie-Tooth News, a recent study has demonstrated the potential impacts of an RNA-based therapy as a treatment for type 1A Charcot-Marie-Tooth disease. The treatment was…
In a recent press release, biopharmaceutical company Swedish Orphan Biovitrum AB ("Sobi") shared that the first patient was dosed in a Phase 3 clinical trial evaluating avatrombopag for pediatric patients…
According to a recent article in the Seattle Cancer Care Alliance, the FDA’s recent approval of Axi-cel (Yescarta) will improve the chances of survival for relapsed or refractory follicular lymphoma.…
It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…
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