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Limb-Girdle Muscular Dystrophy Leaders Advance Collaborative Dialogue to Accelerate Drug Development to Address the Urgent Unmet Need in Limb-Girdle Muscular Dystrophy

/PRNewswire/ On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), convened a.

United statesDouglas sprouleMichelle campbellMatthew wicklundJannie kennedyKatherine mathewsKathryn bryant knudsonNicholas johnsonPeter marksLouise rodino klapacNicole verdunLindsay alfanoPeter kangPeter steinJames signorovitchJerryr mendell

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.

United statesTahseen mozaffarDouglas sprouleKathryn bryant knudsonVikram baliUniversity of californiaDrug administrationSpeak foundationMl bio solutionsBio pharma incBridgebio pharma incSecurities exchangeWorld muscle societyAccelerated approvalBio pharmaNorth star assessment

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