According to a recent article, a new study shows a daily oral dose of Belzutifan may keep patients diagnosed with von Hippel-Lindau disease from needing surgery by shrinking tumors. Von…
According to a recent article, a new study from Boston Children’s Hospital shows that a first-of-its-kind gene therapy technique has succeeded in reversing severe hearing loss from a mutation to…
Givlaari, a treatment for acute hepatic porphyria (AHP), was first approved in Europe back in March of 2020. However, approval doesn't mean immediate access unfortunately. It's been nearly two years…
What does someone with a rare disease look like? With over 7,000 rare diseases in the world, affecting an estimated 300 million people, life with a rare disease (and people…
Positive results have been announced from a Phase 2 trial of olutasidenib, an investigational selective mIDH1 inhibitor used in combination with azacitidine for the treatment of the mIDH1 form of…
The FDA has recently granted Accelerated Approval to Tarpeyo, also called budesonide, to reduce proteinuria in patients with IgA nephropathy (IgAN). Tarpeyo is the first and currently the only treatment…
After several clinical setbacks, Vertex Pharmaceuticals found promising results in its study of a drug to treat a genetic kidney disease called focal segmental glomerulosclerosis (FSGS). As reported recently by…
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