BridgeBio Submits NDA to FDA for BBP-418
If approved, BBP-418 has the potential to become the first and only therapy for individuals living with LGMD2I/R9 and may represent the first approved treatment for any form of...
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Archives
Cure Rare Disease Awarded $7.4 Million CIRM Grant to Advance Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9
Exciting news! https://www.businesswire.com/news/home/20251215136129/en/Cure-Rare-Disease-Awarded-%247.4-Million-CIRM-Grant-to-Advance-Gene-Therapy-for-Limb-Girdle-Muscular-Dystrophy-Type-2iR9...
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BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study
Excellent news for our LGMD2I/R9 family! Link to GlobeNewswire’s article....
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LGMD Scientific Workshop – Article Published
This article is featured in the October 2025 issue of Neurology(R) Clinical Practice:Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development...
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Connecting for a Cure 2024
Check out our Events page for fundraising event details and how you can support this year!...
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