Just saw BridgeBio presented some solid data from their FORTIFY trial at the MDA conference back in March. They're working on BBP-418 for this rare muscle disorder (LGMD2I/R9) and the interim results apparently showed real efficacy. Pretty interesting because there's basically nothing out there for patients with this condition right now.

The FORTIFY study got presented by researchers from Iowa and Yale, which adds some credibility. They even have posters on disease burden, treatment patterns, and the whole translational pathway from lab to clinical trials. That level of detail suggests they're serious about this.

Stock-wise, BBIO was trading around $66 when they presented, up a couple percent that day. Honestly, rare disease biotech is always a gamble, but if this FORTIFY data holds up through Phase 3, could be something worth watching. The fact that they're getting academic collaborators to present alongside the company data is a good sign too.

Anyone else following rare disease plays like this?