The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
The Partnership A new partnership has just been announced between Mendelian, a digital health company for rare disease, and Modality NHS partnership, a rare disease diagnosis program. Together, these organizations…
Neurodegenerative diseases such as Parkinson’s, Alzheimer’s, and multiple sclerosis are disorders that mostly affect the neurons located in the brain. These diseases are incurable, resulting in the death of nerve…
Many people have questions about the COVID-19 vaccine, a number of them pertaining to the speed at which it was developed and approved. Rare disease patients are no exceptions. Barbra…
Toxicity resulting from cancer therapy is usually the result of the repeated administration of cancer drugs. On the other hand, the efficacy of CRISPR-Cas9 has been largely affected by problematic…
According to a recent press release from biopharmaceutical company Praxis Precision Medicines ("Praxis"), two of the company's drug candidates received either Orphan Drug or Rare Pediatric Disease designations for rare…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
A recent announcement by Gilead Company citing data from Kite Pharma, one of its premier companies, set out remarkable results from Kite’s Zuma-1 clinical trial. The findings confirmed that…
As reported in PR NewsWire; in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…
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.
