Shanghai Henlius Biotech announced that the FDA has granted Orphan Drug Designation for HLX22, an anti-HER2 monoclonal ...
Granted Both Orphan Drug & Fast Track designations for Amezalpat (TPST-1120) for the treatment of patients with Hepatocellular Carcinoma ...
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA ...
Although itolizumab did not improve response rates at Day 29 for patients with acute GVHD, the therapy showed significant ...
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SLNO Stock Rallies on FDA Approval of Prader-Willi Syndrome DrugSoleno's shares soar in the premarket hours following the FDA approval of Vykat XR (diazoxide choline) for treating ...
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AstraZeneca’s $1bn hypoparathyroidism drug achieves success at Phase IIIUK-based AstraZeneca’s acquired rare disease drug has met the primary endpoint in a Phase III trial in patients with chronic ...
The FDA has granted orphan drug designation to PEP-010 for pancreatic cancer, advancing its development as a potential novel ...
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 ...
Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030. The 2025 ...
Researchers have found a potential new way to slow the progression of lung fibrosis and other fibrotic diseases by inhibiting ...
Nurix on Monday said the FDA granted orphan-drug designation to bexobrutideg, also known as NX-5948, for the treatment of Waldenstrom macroglobulinemia, a slow growing type of non-Hodgkin's lymphoma ...
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