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Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1

Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1
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United statesCaroline hastingsExchange commissionCompany transportCompany trappsolRafael holdings incGlobal principal investigator for the companyCyclo therapeutics incHealth authoritiesCyclo therapeuticsPaediatric investigational planChief executive officerGlobal principal investigatorRafael holdingsJoshua fineChief financial officer

PepGen Reports First Quarter 2024 Financial Results and Recent Corporate Highlights

PepGen Reports First Quarter 2024 Financial Results and Recent Corporate Highlights
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United statesUnited kingdomJames mcarthurSarah suttonNoel donnellyPepgen incDrug administrationRegulatory agencyInternational myotonic dystrophy consortiumMuscular dystrophy associationDuchenne muscular dystrophyEnhanced delivery oligonucleotideRare pediatric disease designationsOrphan drugDystrophy associationRepeat dose nonclinical data

Satellos Bioscience Announces 2023 Year End Financial Results and Operational Highlights

Satellos Bioscience Announces 2023 Year End Financial Results and Operational Highlights
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New yorkUnited statesCarolyn rogersBlackrock larry finkGabriel friedmanRyan mitchellJoe oconnorFrank gleesonMichael crossPhil lambertJessica yinglingBarbara shecterElizabeth williamsLarry finkLiz williamsVenture company

Edgewise Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

– Overenrolled CANYON and advanced the global pivotal cohort, GRAND CANYON, of EDG-5506 in Becker muscular dystrophy – – Advanced Phase 2 LYNX trial of EDG-5506 including new cohort for boys.

United statesKevin kochDuchenne beckerAmerican heart association scientific sessionsSociety annual scientific sessionsUs food drug administrationSecurities exchangeExchange commissionCardiovascular programEdgewise therapeutics incWorld muscle societyMusculoskeletal programEdgewise therapeuticsChief executive officerNorth star ambulatory assessmentFast track

Biogen s Skyclarys Approved In Europe For Friedreich s Ataxia Treatment

Biogen s Skyclarys Approved In Europe For Friedreich s Ataxia Treatment
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Drug administrationEuropean commissionEuropean unionBiogen incMore such health newsOrphan drugFast trackRare pediatric disease designations