According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…
Hyperphagia is an extreme hunger and obsession with food, and it is a symptom of Prader-Willi syndrome. While many of the other effects of this condition have treatments, this…
Olmsted syndrome is a rare disease most frequently caused by mutations in TRPV3. For some patients, it is caused by MBTPS2 mutations. It is characterized by palmoplantar keratoderma (PPK) as…
-150x150.jpg)
According to a recent article in Pharma Tutor, researchers at NHGRI, a division of the National Institute of Health, identified the biological cause of a newly-discovered autoinflammatory disease they named…
According to a story from Markets Insider, the biopharmaceutical company Principia Biopharma Inc. has recently issued new updates in regards to the timeline of its phase 3 clinical study. This…
The 2019 European Academy of Dermatology and Venereology Congress was held in Madrid, Spain. During this conference, a symposium was held discussing IL-23 inhibition in psoriasis. Here are some of…
“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…
© 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.
