Breakthrough Designation

MetaVia's drug vanoglipel gets a major boost from AI modeling, validating its potential to treat complex metabolic diseases like MASH and Type 2 Diabetes.

An expanded partnership aims to deliver a once-daily oral tablet for hypoparathyroidism, potentially revolutionizing treatment for thousands of patients.

Affinia Therapeutics gets the green light for a human trial of AFTX-201, a one-time therapy for a devastating genetic heart disease, BAG3 DCM.

With crucial data on its novel oral inflammation inhibitor looming, NodThera brings in seasoned leader Geoff McDonough to navigate the path to Phase 3.

New 4-year real-world data shows Zevra's drug stabilizes the rare, fatal 'Childhood Alzheimer's' disease, marking a milestone for patients and families.

As X4 Pharmaceuticals preps for a key investor summit, all eyes are on its pivotal trial for chronic neutropenia and the commercial fate of XOLREMDI®.