Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy
If approved, DTX401 would be the first treatment to address the disease at its root cause
If approved, DTX401 would be the first treatment to address the disease at its root cause
UBT251 appeared to have a safe and well-tolerated profile consistent with incretin-based therapies
The agreement will see the two companies jointly develop and commercialise premium nutraceutical ingredients in advanced transdermal delivery formats
Vanrafia, a potent endothelin A receptor antagonist, received accelerated approval in the US and China in 2025 for reducing proteinuria in adults with IgAN
The paper highlights how conditions common across India—including anemia, inherited hemoglobin disorders, and glucose-6-phosphate dehydrogenase (G6PD) deficiency—can significantly distort HbA1c readings
The Phase III programme, Enith1 and Enith2, is expected to launch this quarter
ALV-100 is a bifunctional fusion protein combining glucose-dependent insulinotropic polypeptide receptor (GIPR) antagonism with glucagon-like peptide-1 receptor agonism
The trial’s primary endpoint is the mean change in HbA1c from baseline, while secondary endpoints include changes in fasting blood glucose, body weight, and overall safety
Fasting glucose improved by 12.3 mg/dL, while liver stiffness dropped 23.7%, signaling a direct hepatic benefit
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