“I feel like I’m in a huge dream that nobody can get into,” Noemie explained, describing her weeks of intensive sleep during which she slept 20 hours a day, the…
Marinus Pharmaceuticals has recently announced updates on its ganaxolone programs for tuberous sclerosis complex (TSC), PCDH19-related epilepsy (PCDH19-RE), and CDKL5 deficiency disorder (CDD). Ganaxolone Ganaxolone is a positive allosteric modulator…
Marinus Pharmaceuticals is a pharmaceutical company that works to create treatments for epilepsy and other neuropsychiatric disorders. They are in the process of conducting clinical trials to evaluate the safety…
-150x150.jpg)
Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the European Medicines Agency’s (EMA) Committee for…
-150x150.jpg)
According to a recently published article in Targeted Oncology, the results of the Phase III CANDOR trial were presented at the 61st ASH annual conference in Orlando, Florida. The…
-150x150.jpg)
A recent article published in Globe Newswire heralded Marinus Pharmaceuticals’ new drug, ganaxolone. The drug has been studied in over 1600 patients and is now being tested in two clinical…
-150x150.jpg)
At times there seems to be no happy medium in life. A recent article in Bloomberg claims that the speed at which the FDA is approving newly developed drugs is…
Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
