According to a story from Market Screener the biotechnology company Bioblast Pharma has announced a new partnership with Team Sanfilippo a nonprofit foundation which is committed to medical research related…
The Food and Drug Administration (FDA) recently announced its approval of Venetoclax (Venclexta) in combination with either decitabine or azacitidine (hypomethylating agents) or cytarabine (an antimetabolite) to treat adults 75 years…
“Doctor” Onelio Hipolit-Gonzalez has been charged with unlicensed practice of a health care profession and unlawful use of a two-way communication device according to an article published in the Spectrum News of…
Ocugen Pharmaceuticals has just received their first ever orphan drug designation by the FDA for a gene therapy treatment. It's called OCU400 and it's being investigated as a treatment for…
Cystic Fibrosis Cystic fibrosis (CF) is a rare disease caused by the lack (or malfunctioning) of the cystic fibrosis transmembrane conductance regulator (CTFR) protein. This occurs when children inherit a mutated…
Neutropenia Neutropenia is a rare condition that affects approximately 1 in every 200,000 newborns. It results in a reduced number of neutrophils in the body. Neutrophils are white blood cells,…
A report published in 2018 in The Hemotologist mentions the fact that most of the therapies introduced over the years for acute myeloid leukemia (AML) simply meant optimizing the use of…
According to a story from globenewswire.com, the biopharmaceutical company Lysogene and the drug developer Sarepta Therapeutics, Inc., have recently announced that the first patient has been dosed in a Phase…
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.
