Presentations & Publications

PRESENTATION · Mar 2025 · PDF

BBP-418 - MDA 2025 - Understanding the Burden of LGMD2I/R9

PRESENTATION · Mar 2025 · PDF

BBP-418 - MDA 2025 - Baseline Characteristics FORTIFY

PRESENTATION · Dec 2024 · PDF

BBP-418 - WMS 2024 - FORTIFY Design

PRESENTATION · Dec 2024 · PDF

BBP-418 - MENA 2024 - Oral Presentation

PRESENTATION · Dec 2024 · PDF

BBP-418 - MDA 2024 - FORTIFY Phase 3 Trial Design

PRESENTATION · Dec 2024 · PDF

BBP-418 - MDA 2024 - FORTIFY Phase 3 Trial Design

PUBLICATION · Mar 2024 · PDF

Journal of Muscle Research and Cell Motility, Validation of a Novel Western Blot Assay to Monitor Patterns and Levels of Alpha Dystroglycan in Skeletal Muscle of Patients with Limb-girdle Muscular Dystrophies

PRESENTATION · Dec 2023 · PDF

BBP-418 - WMS 2023 - Phase 2 Data at 21 Months; Ambulatory Improvements at 9 Months

PRESENTATION · Dec 2023 · PDF

BBP-418 for LGMD2I/R9 - MDA 2023 - Oral Presentation - Phase 2 Data at 15 Months

PRESENTATION · Dec 2023 · PDF

BBP-418 for LGMD2I/R9 - MDA 2023 - Poster - Phase 2 Data at 15 Months

PRESENTATION · Dec 2023 · PDF

BBP-418 for LGMD2I/R9 - MDA 2023 - Preliminary Data on Bioassay Measuring αDG

PRESENTATION · Dec 2023 · PDF

BBP-418 for LGMD2I/R9 - Novel Bioassay, Phase 2 Data and Phase 3 Trial Design in LGMD2I

PUBLICATION · Dec 2022 · PDF

PLOS One, Ribitol Dose-dependently Enhances Matriglycan Expression and Improves Muscle Function with Prolonged Life Span in Limb-girdle Muscular Dystrophy 2I/R9 Mouse Model

PUBLICATION · Aug 2018 · PDF

Nature Communications, Ribitol Restores Functionally Glycosylated Alpha-dystroglycan and Improves Muscle Function in Dystrophic FKRP-mutant Mice