uniQure Announces New Preclinical Data in Hemophilia A and Fabry Disease in Oral Presentations at the 22nd ASGCT Annual Meeting
May 02, 2019 07:00 ET
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uniQure Inc.
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 02, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with...
uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT)
April 15, 2019 12:15 ET
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uniQure Inc.
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 15, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with...
uniQure Highlights Pipeline Expansion and Advancements in Technology at Research & Development Day
November 19, 2018 12:15 ET
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uniQure Inc.
~ Unveils New AAV Gene Therapy Approaches to Hemophilia A, Fabry Disease and Spinocerebellar Ataxia Type 3 ~ ~ Introduces miQURE™ Gene Silencing Platform with Applications Across Multiple Indications...
Rare Hematology Market to Represent a Value of over US$ 16,000 Million by the End of 2022: Transparency Market Research
November 14, 2017 03:14 ET
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Transparency Market Research
Albany – NY, Nov. 14, 2017 (GLOBE NEWSWIRE) -- Growing prevalence of blood disorders has led to an upsurge in demand for the rare hematology globally. However, limited resources and disease-specific...
UK Regulatory Agency Approves Continued Enrollment in BioMarin Phase 1/2 Study of BMN 270 in Hemophilia A
October 13, 2016 16:01 ET
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BioMarin Pharmaceutical Inc.
Phase 1/2 Study Expected to Resume by End of 2016 Requirement for Prophylactic Corticosteroids Removed SAN RAFAEL, Calif., Oct. 13, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc....
BioMarin Provides Positive Proof-of-Concept Data for BMN 270 Gene Therapy in Hemophilia A in Late Breaking Oral Presentation at the World Federation of Hemophilia (WFH) 2016 World Congress
July 27, 2016 14:30 ET
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BioMarin Pharmaceutical Inc.
6 of 7 High Dose Patients Show Factor VIII levels above 50%, 7th patient above 10% No Clinically Relevant Sustained Rises in ALT Phase 2b Study to Begin Mid-2017 for Potential Accelerated Approval...
Catalyst Biosciences and CMC Biologics Announce Manufacturing Agreement for Catalyst’s Next-Generation Factor VIIa Product CB 813d
May 24, 2016 08:30 ET
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Catalyst Biosciences, Inc.
- Companies Committed to Establishing Manufacturing Capabilities for Next-Generation Factor VIIa CB 813d -- Catalyst Plans to Initiate Clinical Pivotal Trial in Hemophilia A and B “Inhibitor” Patients...
BioMarin Provides Encouraging Preliminary Data on First 8 Patients in Hemophilia A Gene Therapy Program
April 20, 2016 08:30 ET
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BioMarin Pharmaceutical Inc.
Two High Dose Patients Show Increasing Levels of Factor VIII Above 50% Five of Six High Dose Patients Show Factor VIII Levels Above 5% SAN RAFAEL, Calif., April 20, 2016 (GLOBE NEWSWIRE) -- ...
BioMarin Receives European Orphan Drug Designation for BMN 270, First Investigational AAV-Factor VIII Gene Therapy for Patients with Hemophilia A
March 24, 2016 08:30 ET
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BioMarin Pharmaceutical Inc.
SAN RAFAEL, Calif., March 24, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that BMN 270, an investigational gene therapy for the treatment of hemophilia A,...
BioMarin Receives Orphan Drug Designation From FDA for First AAV-Factor VIII Gene Therapy, BMN 270, for Patients With Hemophilia A
March 01, 2016 17:42 ET
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BioMarin Pharmaceutical Inc.
SAN RAFAEL, Calif., March 01, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that BMN 270, an investigational gene therapy for the treatment of patients with...