For Part 2 of "Save Our Medicine," we continue talking with Tiffany, DeAnna, and Sara about their families' NPC journeys, as well as the push for medication access. If you…
According to a recent article, the nonprofit Gilbert Family Foundation has offered to fund the $5.4 million clinical study to better understand vision loss from NF1-associated optic pathway gliomas (NF1-OPGs).…
To learn more about Rachael's story, Niemann-Pick type C (NPC) symptoms, the diagnostic process, and how her mother Debbie found and offered support to other families, take a look at Parts…
According to a November 23 article from Muscular Dystrophy News, the FDA recently granted Orphan Drug designation to clinical-stage biotechnology company Regenxbio's experimental gene therapy candidate, RGX-202. The gene therapy,…
Jeffrey Miner, Ph.D., Director of Basic Research in Washington University’s Division of Nephrology, has received a $2.25M five-year grant from the National Institute of Diabetes and Digestive and Kidney…
A recent poster presentation at the 2021 NANETS Annual Meeting focused on the RELAX study, which evaluated patient-reported outcomes from carcinoid syndrome (CS) patients being treated with telotristat ethyl. According…
Jo Traunter was told that she was "a bleeder" throughout her life, whether it was by family, doctors, or others. She earned this title after living through decades of heavy…
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