The FDA estimates that there are about five hundred people in the USA with the SOD1 mutation out of a total of approximately thirty thousand ALS patients. According to a…
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…
KMT2A rearrangements—a DNA fault—lead to treatment-resistant acute lymphoblastic leukemia (ALL) in infants. While around 50% of children with KMT2A rearrangements respond well to intensive chemotherapy, the remaining 50% do…
You caught me: I'm a Swiftie. I first saw Taylor Swift on her Speak Now tour and, in 2 weeks, am taking on the Eras tour in Philadelphia (PS: if you have any…
There is no cure for cystic fibrosis. That is the reason the Cystic Fibrosis Research Institute (CFRI) is very vocal and makes it clear that cooperation is needed from the…
Throughout the globe, there are many countries in which rare disease awareness and support are still lacking. While India has made some strides in improving care for people within…
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