Currently, the standard-of-care for individuals living with hemophilia B is intravenous factor IX infusions (prophylaxis). These infusions can be time-consuming, require adherence to a strict schedule, and may not…
According to a story from CNN, a new treatment was recently approved by the US Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's…
In July 2022, Global Genes, a nonprofit 501(c)(3) organization and global advocate for those with rare diseases, awarded its Health Equity in RARE Patient Impact grants to members of…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. These are defined as conditions affecting fewer…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, we discussed Dr. Pariser's background, what hyperhidrosis is, and its symptoms and manifestations. Today, we'll…
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