At the end of May, 2022, the Governor of Connecticut, Ned Lamont, established a permanent Rare Disease Advisory Council (RDAC) for the state by signing House Bill 5500 (HB 5500).…
When Lauren Atkins and William Bromley welcomed their son Charlie into their lives, they were thrilled. Jessie, their daughter, may have been even more thrilled, already having a soft spot…
The FDA has just announced that they have lifted the temporary hold placed on pheNIX gene therapy. This gene therapy was created by Homology Medicine as an investigational treatment for…
The FDA has recently approved combination treatments for those diagnosed with unresectable advanced or metastatic esophageal squamous cell carcinoma. These combination treatments are nivolumab and fluoropyrimidine along with platinum-containing chemotherapy…
According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…
In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which…
Recently diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations now have a new treatment option, as the FDA has granted approval to TIBSOVO (ivosidenib tablets)! This is an expansion…
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