Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they…
The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide is effective in the treatment…
Researchers at Washington University’s medical school have used a new technique to study brain neurons in a lab setting, allowing them to bypass the need to rely on a brain…
Clinical trials play a crucial role in better understanding rare diseases and therapeutic interventions. According to a news release from rare disease company Albireo Pharma, Inc. (“Albireo”), enrollment is now…
According to a recent article from Physician’s Weekly, two trials recently published are showing the results of striving to improve the management and outcomes in nonalcoholic steatohepatitis (NASH) and…
According to a recent article, the nonprofit Gilbert Family Foundation has offered to fund the $5.4 million clinical study to better understand vision loss from NF1-associated optic pathway gliomas (NF1-OPGs).…
Every year, actually every day, the FDA and similar organizations for other countries approve new medicines. Innumerable pharmaceutical companies work tirelessly for years to research, develop, test, distribute, and market…
I revisited an article about a clinical trial that focused on women with pulmonary arterial hypertension (PAH), and I’m still baffled, because it just doesn’t make sense. The information provided about…
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