"Cellenkos gains FDA orphan drug status for CK0804 to treat myelofibrosis" was originally created and published by ...

CGTxchange leverages the combined capabilities of the organizations in an unprecedented way to engage a broad array of potential partners and funders to efficiently identify, evaluate, finance, ...

Rilzabrutinib is an investigational, oral reversible Bruton tyrosine kinase inhibitor. The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of ...

Treg therapeutic designed to preferentially traffic towards its ligand CXCL12 over expressed in the bone marrow and spleen in myelofibrosis. -- CK0804 engages with antigen presenting cells within ...

Most orphan drug spending does not actually go to patients with rare diseases, a new study shows. Pharmaceutical companies creating and launching orphan drugs for rare diseases do so under a landmark ...

Every day, someone in the U.S. is born with or wakes up to unexplained symptoms and the subsequent diagnosis of a serious illness. If it’s a disease one has heard of, like diabetes, heart failure or ...

Buerger disease typically affects young or middle aged male cigarette smokers. The Food and Drug Administration (FDA) has granted Orphan Drug designation to AMB-301 for the treatment of Buerger ...

Peter L. Salgo, MD: Let’s broaden this out just a little bit, because Huntington is a rare disease, but there are lots of rare diseases out there. How would you define a rare disease? Is there a ...

An anopheline mosquito taking a blood meal. Researchers and physicians, open your guides to rare diseases, for that may be the only place you'll encounter Jumping Frenchmen of Maine. There you also ...