BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set
On 6ix: 6ix.com/news/biomarin-announces-positive-phase-3-gene-therapy-trial-results-in-adults-with-severe
Watch BioMarin Pharmaceutical's latest callBioMarin Pharmaceutical (NASDAQ:BMRN) Q2 2026 Earnings Call · Replay · Aug 6Read the transcript of BioMarin Pharmaceutical (NASDAQ:BMRN) Q2 2026 Earnings CallBioMarin Pharmaceutical Inc. on 6ix
Story so far
- BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection for Treatment of Adults with PKU
- BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
- BioMarin Provides Additional Data from Recent 4 Year Update of Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit
Earlier from BioMarin Pharmaceutical
- BioMarin Announces Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study
- BioMarin and Deep Genomics to Collaborate on Advancing Programs Identified Using Artificial Intelligence
- BioMarin Expands Vosoritide Clinical Program
- BioMarin Announces Third Quarter 2020 Total Revenues of $477 Million
- BioMarin to Participate in the Credit Suisse 29th Annual Virtual Healthcare Conference on November 10 at 12: 30pm ET
Later from BioMarin Pharmaceutical
- BioMarin to Host Fourth Quarter and Full Year 2020 Financial Results Conference Call and Webcast on Thursday, February 25 at 4: 30pm ET
- BioMarin Earns Top Marks in Human Rights Campaign's 2021 Corporate Equality Index
- BioMarin Appoints Former Goldman Sachs Partner and Life Sciences Executive, Maykin Ho, Ph.D. to Board of Directors
- BioMarin Announces Fourth Quarter and Record Full-year 2020 Financial Results and Corporate Updates
- BioMarin Completes Full Enrollment in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
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