When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting…
Near the end of September, Hansa Biopharma ("Hansa") announced promising data from a Phase 2 clinical trial on imlifidase. This therapy is designed to treat patients with severe anti-GBM antibody…
For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…
If you're like me, you might be surprised that COVID-19 is not only still deeply ingrained in our world, but it seems to continue growing. As of the afternoon of…
At the start of October, Swedish-based IRLAB announced that the FDA approved an investigational new drug application (IND) for their therapeutic candidate mesdopetam (IRL790). This therapy is designed to treat…
According to a press release, biopharmaceutical company Apellis Pharmaceuticals ("Apellis") recently launched registration programs to explore pegcetacoplan for patients with C3 glomerulopathy (C3G), amyotrophic lateral sclerosis (ALS), and immune…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
The FDA has recently granted the Breakthrough Therapy designation to FARXIGA, a chronic kidney disease treatment created by AstraZeneca. This designation will allow for a quicker development, which will hopefully…
The global biopharmaceutical company Incyte recently announced the first-ever recipient of its Incyte Ingenuity Award, which will be the Massachusetts General Cancer Center (MGCC). The purpose of this award is…
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.
