The therapy shows efficacy in improving diarrhea, abdominal pain in GLP-1, GLP-1/GIP-treated patients with diabetes and/or obesity in a small study.

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

The trial will examine the efficacy, safety, and pharmacodynamics of S-606001 SRT used with ERT in confirmed LOPD adults.

Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a ...

Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter "Shionogi") announced the first patients were enrolled in Esprit, a global Phase 2 clinical ...

Sanofi’s venglustat earns Breakthrough Therapy designation in the US for type 3 Gaucher diseaseParis, March 18, 2026. The US Food and Drug ...

Novartis has committed up to $3 billion—two-thirds of that upfront—toward acquiring Pikavation Therapeutics, a subsidiary of Synnovation Therapeutics that specializes in developing PI3Kα inhibitor ...

The U.S. Food and Drug Administration has granted breakthrough therapy designation to Sanofi’s (SNY) venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the ...

US FDA grants Breakthrough Therapy designation to Sanofi’s venglustat for type 3 Gaucher disease: Paris Thursday, March 19, 2026, 10:00 Hrs [IST] Sanofi announced that the US Fo ...

Chronic pancreatitis is now recognized as a complex fibroinflammatory disorder involving genetic, environmental, and metabolic factors.

Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines in areas of high unmet need in rare muscle diseases, ...

Read more about the newly discovered molecule, SU212, that impedes the growth of triple-negative breast cancer.