It can be extremely difficult to incentivize drug development for rare diseases or conditions. To combat this, decades ago, the FDA created the Orphan Drug Act. Now, Orphan Drug designation…
Before you read on, make sure to check out Part 1 of our interview where we discuss what SLC6A1 is and Aubrey's diagnostic journey. Today, we'll be discussing what it's like to…
According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…
In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…
Before you read on, make sure to check out Part 1 of our interview. In Part 1, we discussed Dr. Sumeray's background, what hypoparathyroidism is, and its symptoms and treatments. Today, we're…
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