Since the dawn of time, the human body has changed to cope with microbes and environmental changes. As a result, specific gene mutations evolved to protect populations from harm.…
In 2019, Alnylam Pharmaceuticals received FDA approval for Givlaari, the first RNAi therapy for patients with acute hepatic porphyria. Although some were initially worried about the therapy's safety and…
According to a story from BioSpace, the drug company Zynerba Pharmaceuticals Inc. recently released the results from a clinical trial. This trial was testing the company's product Zygel™ (CBD gel)…
David Fajgenbaum toes the line between patient and doctor. After growing up with a physician as a father, Fajgenbaum chose to follow in his footsteps. His most important lessons came…
In 2016, 6-year-old Alec Ahmad loved club soccer, playing games with his two brothers, and running around with his friends at recess. He was full of energy. But every once…
A cannabinoid medication that uses a non-psychoactive ingredient of cannabis to treat rare forms of epilepsy has now been recategorized as a schedule 5 drug in the UK, the least…
At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
If you're experiencing a difficult time with COVID-19, you're not alone. Many people, including myself, are struggling with managing anxiety, stress, and isolation. Additionally, this time period can feel traumatic,…
Vision loss is a major symptom of Stargardt disease, so a main goal of treatments is to combat this loss. In order to test the effectiveness of these treatments, there…
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