Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

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

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant ...

BioNTech's gotistobart receives FDA orphan drug designation, showing promise in improving survival for patients with squamous ...

NP-G2-044 (Prilukae), a novel oral fascin inhibitor, has earned orphan drug designation (ODD) from the FDA for the treatment ...

Quince Therapeutics (QNCX) rated Buy ahead of Phase 3 NEAT readout in A-T; eDSP targets $1B+ orphan market with strong cash ...

AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. For more information, ...

Arbele, a biotechnology company focused on developing innovative targeted cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) ...

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 ...

Rising concerns about spending on prescription drugs that treat rare diseases are not justified, according to a new analysis in the journal Health Affairs. "We wanted to focus on the true impact of ...

The One Big Beautiful Bill Act takes a big step in the rare disease space by expanding the contours of the orphan drug exclusion, a once narrow exception that permitted manufacturers of rare disease ...