About HAE Hereditary angioedema (HAE) is a rare disease that is caused by the deficiency or reduction of a protein called C1-INH in the body. When this protein is not present,…
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Scientists believe that “it is in our DNA.” According to a recent article in EurekAlert, a five-year study formulated through Open Targets together with the Sanger Institute and their…
According to a press release from Barcelona-based biotechnology company Minoryx Therapeutics, the Company has completed enrollment for a phase 2 clinical study of its experimental Friedreich's ataxia treatment, MIN-102 (generic…
As originally reported in SF Gate, Jeannette and Victor Vega quickly realized that taking care of their second daughter, Tiana, was not going to be so easy this time. From…
Sarah experienced her first symptoms at age eight. She suffered frequent and long migraines, despite being so young. Doctors were concerned she may have meningitis but never got to the…
According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…
According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…
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Lately, there have been many newsworthy articles about Novartis and Zolgensma (onasemnogene abeparvovec-xioi). In May 2019 the FDA approved Zolgensma, a one-time treatment for the most severe form of…
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