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Bridgebio Pharma Presents 12-Month P-II Study Results of BBP-418 for Limb-Girdle Muscular Dystrophy Type 2i at WMS 2022

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Bridgebio Pharma Presents 12-Month P-II Study Results of BBP-418 for Limb-Girdle Muscular Dystrophy Type 2i at WMS 2022

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  • The P-II study evaluating the safety, tolerability, feasibility, and usefulness of selected clinical efficacy & PD of BBP-418 in 14 patients (incl. ambulatory & non-ambulatory) with LGMD2i
  • The results showed increased glycosylation of ⍺DG in all dose cohorts with an avg. increase in ⍺DG ratio of +0.21 @Day 90, ≥75% reduction in CK sustained @12mos., improvements from baseline in NSAD (0.95) & 10MWT velocity (0.09m/s) @12mos. with no treatment-related SAEs or dose-limiting toxicities, reduction in muscle damage & motor function
  • BridgeBio & ML Bio Solutions closely work with regulatory agencies & will initiate a P-III trial in 2023. BridgeBio also highlighted the development of a novel bioassay to evaluate the extent of ⍺DG glycosylation from muscle biopsy samples

Ref: Bridgebio Pharma | Image: Bridgebio Pharma

Click here to­ read the full press release 

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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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