According to Healio, the U.S. Food and Drug Administration (FDA) recently granted Fast Track designation to LioCyx-M004. This therapy, developed by T-cell immunotherapy company Lion TCR, is designed to…
WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
In the European Union (EU), Orphan Drug designation is given to drugs or biologics intending to improve the treatment, diagnosis, or prevention of rare or serious illnesses. To be…
Continued From Part One Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering.…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this status, “rare” conditions…
ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in the…
Clinical trial results can help support drug developers' goals. For example, positive trial results may support Marketing Authorization Applications (MAAs) or other areas needed to get therapies into the…
January has been designated as a time to bring awareness to alternating hemiplegia of childhood! Alternating hemiplegia of childhood is extremely rare, with about 1 in 1,000,000 reported cases; therefore…
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.
