According to a recent press release from Passage Bio, their investigational gene therapy for Krabbe disease, PBKR03, may be ready to move into the clinical trial stage of drug development.…
To be able to pursue your dreams and passions is nothing short of empowering. But for many people within the rare disease community, there are barriers to pursuing our goals.…
In the years leading up to menopause (perimenopause), migraines and headaches become increasingly common. Pair this with mental fogginess and memory issues, changes in mood, altered eyesight, sleep difficulties, and…
In 1873, a Russian physician coined the term “multiple myeloma” after discovering a patient with eight different forms of bone marrow tumors. In the early years, patients were treated with…
Bristol Myers Squibb (BMS) has been developing luspatercept-aamt - marketed as Reblozyl - as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license…
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…
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