During a recent WZDX News interview with Dr. David Bick, HudsonAlpha Faculty Investigator Dr. Bick stated that in Alabama alone, there are approximately four hundred thousand people with rare diseases.…
The Brazilian Health Regulatory Agency, ANVISA, has granted market approval to Insightec for their focused ultrasound, Exablate 4000. This MR-guided ultrasound is used for the treatment of tremors that define…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from azfamily.com, Reyna Felix, age 28, was at work as a 911 dispatcher when her coworkers said that she started behaving strangely. Around midnight, she fell…
An estimated 1 in 10 Americans, or 1 in 20 people globally, have or will have a rare disease at some point. Rare Disease Day takes place on February 28. Ultimately,…
According to a February 25 press release, Amondys 45 (casimersen), a therapeutic option for patients with Duchenne muscular dystrophy (DMD), recently became FDA-approved. The therapy is specifically designed for patients…
On February 18, 2021, specialty pharmaceutical company Kyowa Kirin Co., Ltd. ("Kyowa Kirin") shared that a Phase 2 clinical trial evaluating KHK4083 had reached its primary endpoint. KHK4083 is an…
According to BioSpace, Biohaven Pharma has utilized their MATE platform to create and develop a hyperimmune globulin mimic (HGM) that is able to both bind to and neutralize various strains…
Billions of dollars have been spent in research over decades, resulting in only two therapies that treat symptoms of Alzheimer’s disease (AD). There are no approved methods that diminish the…
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