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BridgeBio Pharma Shares Positive Long-Term Data from an Ongoing Phase 2 Study, which Support the Potential Use of Glycosylated Alpha-dystroglycan (⍺DG) Levels as a Surrogate Endpoint in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

- Early assessment of increased glycosylated ⍺DG levels at 3 months predicted subsequent ambulatory improvements at 9 months, supporting the use of glycosylated ⍺DG levels as a potential surrogate. ....

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Hull man with life-limiting muscular disease hoping he can keep playing Powerchair football - but he needs help

Hull man with life-limiting muscular disease hoping he can keep playing Powerchair football - but he needs help
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Szymczak siblings raise over $1,600 through lemonade stand for Make-A-Wish

RACINE COUNTY The Szymczak siblings tallied in over $1,600 for Make-A-Wish Wisconsin through a lemonade stand that they hosted this past weekend in Caledonia. ....

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BridgeBio Pharma Announces Opportunity for Accelerated Approval Pathway in Limb-girdle Muscular Dystrophy Type 2I (LGMD2I/R9) Based on Glycosylated Alpha-dystroglycan (⍺DG) Levels and Announces First Patient Dosed in FORTIFY Phase 3 Study

- BridgeBio met with the U.S. Food and Drug Administration to discuss the use of glycosylated αDG levels as a surrogate endpoint; based on this meeting, the Company believes there is potential to. ....

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AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD)

LGMD is a Rare Form of Muscular Dystrophy with No Approved Therapy RESEARCH TRIANGLE PARK, N.C., Feb. 27, 2023 /PRNewswire/ Asklepios BioPharmaceutical, Inc. (AskBio), a wholly ....

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