According to a press release put out by PTC Therapeutics, Inc., the Office of Drug Evaluation of the Food and Drug Administration on Wednesday, Oct. 25th, 2017, sent a complete…
Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…
There's a new treatment for X-Linked Hypophosphatemia (XLH) that may be available in the not-so-distant future. XLH occurs when the kidneys process phosphate abnormally, leading to excess phosphate loss in…
There's an app for everything right? It seems like every day a new app is being created to make life easier, for better or for worse. In the case of…
Lucy Babcock is in her fourth year in college and so close to acquiring that diploma and pursuing grad school. Most people suffer from senioritis but Lucy is battling out…
For patients, caregivers, healthcare providers, and anyone interested in learning more about liver diseases, there is a website called Focus. This website is organized by CLDF, which stands for Childhood…
In the ultimate feat of recycling, scientists at UCLA and the Howard Hughes Medical Institute have re-purposed the LeXis gene as a cholesterol-lowering agent. A recent press release issued by…
Over the years, I've talked to dozens of people with Parkinson's disease (PD). They come from all socio-economic backgrounds. Rich and poor. Male and female. But the one thing they…
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