According to a story from Healio, Dr. Monica Konerman recently gave a presentation about the importance of lifestyle changes in treating nonalcoholic fatty liver disease and its rarer and more…
The companies Genentech and Roche are working together to carry out a program of studies involving patients with Huntington’s disease (the huntingtin-lowering trial ASO program). One is a natural history…
This is a video presentation I created called Living Well with Dystonia. It shares some of the many things I have learned in my almost 20 years of living with…
The United States Food and Drug Administration has granted the drug Lynparza® (olaparib) Orphan Drug Designation for the treatment of pancreatic cancer. Lynparza is used in the US to treat…
You may know her from movies like Legally Blonde, Cruel Intentions, and Hellboy - but now Hollywood star Selma Blair has a new role she's tackling: multiple sclerosis advocate. Just…
The United States Food and Drug Administration has granted Fast Track designation to a drug being researched as a potential treatment for progressive familial intrahepatic cholestasis, a serious liver disease…
Across the globe, countries are transitioning their healthcare systems to provide more care within the patient's home and less in a hospital setting. Why? Simply put, it's because there's beginning…
British Columbia faces excruciating decisions about how and whether to cover drug treatment for rare diseases. One particular drug is Spinraza. It is a drug that is a part of…
The United States Food and Drug Administration has awarded Rare Pediatric Disease Designation to a potential drug for Mucopolysaccharidosis type I. For more detailed information about this news, you can…
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