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Just saw BridgeBio is presenting more Phase 3 FORTIFY trial data at the MDA conference this month. They're showing results for BBP-418 in LGMD2I/R9 patients - pretty rare genetic muscle disorder stuff. Katherine Mathews from University of Iowa is doing the main presentation, and apparently Yale's team is also showing up with research on measuring ribitol response across different FKRP variants, which sounds like it could be important for understanding how the treatment works across different patient mutations.
They've got four posters too covering disease burden and long-term survival modeling. Stock's been bouncing around between $28 and $84 over the past year, closed at $66.54 last time I checked. What's interesting is how much these rare disease plays move on clinical data - the market seems to be paying attention to this one. Not sure if the interim data will be the catalyst people are hoping for, but the fact they're presenting at a major conference suggests they think there's something worth sharing.