Neuren Pharmaceuticals has announced it will develop a new drug to treat a type of life-threatening brain injury in infants, ...

Net loss for the full year ended December 31, 2024 was $43.3 million, compared to $29.5 million during the comparable period in 2023, primarily reflecting an operating loss of $47.4 million, partially ...

Initiated IND-enabling toxicology program IND submission planned for 4Q25; Clinical data expected in 1H26 CRANBURY, N.J., March 25, 2025 /PRNewswire/ -- Palatin ...

AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).

The FDA granted an orphan drug designation to PEP-010 for the treatment of patients with metastatic pancreatic cancer.

Rhythm Pharma gets orphan drug designation for its setmelanotide to treat hypothalamic obesity in Japan: Boston Friday, March 21, 2025, 16:00 Hrs [IST] Rhythm Pharmaceuticals, Inc ...

The first time Dr. Fajgenbaum repurposed a drug, it was in an attempt to save his own life. At 25, while in medical school, ...

First patient dosed in Phase II OCULE-01 study with roginolisib, an allosteric modulator of PI3KdThe European Medicines Agency (EMA) has granted Orphan Drug Designation for roginolisib for treatment ...

Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to treat hyperphagia and obesity. Rhythm is evaluating setmelanotide in a global Phase 3 trial in patients with acquired hypothalamic ...

Rhythm Pharmaceuticals (RYTM) announced it has received orphan drug designation from Japan’s Ministry of Health, Labour and Welfare for ...

Rhythm Pharmaceuticals received an orphan-drug designation from Japan's Ministry of Health, Labour and Welfare for its treatment for a certain type of obesity.