Ultragenyx Pharmaceutical Inc.

205 articles with Ultragenyx Pharmaceutical Inc.

• Ultragenyx Announces UX007 Granted Fast Track Designation and Rare Pediatric Disease Designation by U.S. FDA for Treatment of Long-Chain Fatty Acid Oxidation Disorders

  4/16/2019

  Company on Track to Submit NDA to FDA in Mid-2019

• Ultragenyx to Present Corporate Update at Analyst and Investor Day on April 17 in New York

  4/11/2019

  Ultragenyx Pharmaceutical Inc. announced that management and external experts will provide an update to the investment community on the global commercial launch of Crysvita®, the company’s gene therapy programs and platform including manufacturing capabilities, and preclinical data on the next three Investigational New Drug applications the company plans to submit to the U.S. Food and Drug Administration.

• Ultragenyx Announces Approval of Crysvita® (burosumab) in Brazil for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children

  3/26/2019

  First Latin American Approval of Crysvita, the only Treatment that Targets the Underlying Cause of this Rare, Hereditary, Lifelong Disease

• Ultragenyx to Present at Upcoming March 2019 Investor Conferences

  3/11/2019

  Ultragenyx Pharmaceutical Inc. today announced that it will present at the following upcoming investor conferences:  

• Ultragenyx Announces Proposed Public Offering of Up to $250,000,000 of Its Shares Common Stock

  2/25/2019

  Ultragenyx Pharmaceutical Inc. announced that it has commenced an underwritten public offering of up to $250,000,000 of shares of its common stock.

• Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

  2/21/2019

  DTX401 Response in Time to Hypoglycemia and Improved Glucose Control Maintained or Improved in All Three Patients

• Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update

  2/19/2019

  Strong Launch Continues With More than 550 Patients on Reimbursed Commercial Crysvita® (burosumab) Therapy in the United States at End of 4th Quarter 2018, 80 Percent Increase Versus End of 3rd Quarter

• Ultragenyx and Kyowa Kirin Announce Positive 64-Week Results for Crysvita® (burosumab) from Phase 3 Study in Children with X-linked Hypophosphatemia (XLH)

  2/14/2019

  Results Confirm and Extend 40-Week Findings that Treatment with Crysvita is Superior to Conventional Therapy

• Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2018 Financial Results and Corporate Update

  2/12/2019

  Ultragenyx Pharmaceutical Inc. announced that it will host a conference call on Tuesday, February 19, 2019 at 5pm ET to discuss its financial results and corporate update for the fourth quarter and the year ended December 31, 2018.

• Ultragenyx Appoints Shehnaaz Suliman, M.D., to Board of Directors

  2/4/2019

  Ultragenyx Pharmaceutical Inc. announced the appointment of Shehnaaz Suliman, M.D., to the company’s Board of Directors, effective January 30, 2019.

• Ultragenyx Announces Positive Topline Data from Ongoing Long-Term Extension Study of UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders

  1/22/2019

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced positive topline data from an ongoing long-term extension study of UX007 in patients with long-chain fatty acid oxidation disorder (LC-FAOD), demonstrating sustained reductions in the duration and frequency of major clinical events (MCE) and a long-term safety profile similar to what has previously been seen with UX007.

• Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

  1/4/2019

  DTX401 Response Observed in All Three Patients, with Two Patients Demonstrating Clinically Meaningful Improvement in Time to Hypoglycemia

• Ultragenyx to Present at the 37th J.P. Morgan Healthcare Conference

  1/3/2019

  Ultragenyx Pharmaceutical Inc. today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 37TH Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2019 at 4:00 pm PT in San Francisco.  

• ltragenyx and Kyowa Kirin Announce Health Canada Approval of Crysvita™ (burosumab injection) for the Treatment of X–linked Hypophosphatemia (XLH) in Adults and Children

  12/6/2018

  First Approved Treatment for XLH in Canada that Targets the Underlying Cause of this Rare, Hereditary, Lifelong Disease

• Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Mid-2019

  11/14/2018

  Ultragenyx Pharmaceutical Inc. today announced that it has completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) and plans to submit a New Drug Application (NDA) to the FDA for UX007 for the treatment of patients with long-chain fatty acid oxidation disorders (LC-FAOD) in mid-2019.  

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  BioSpace Movers and Shakers Nov. 12

  11/12/2018

  Biopharma companies name new members of their leadership teams. Who made big moves this week?

• Ultragenyx to Present at Upcoming November 2018 Investor Conferences

  11/9/2018

  Ultragenyx Pharmaceutical Inc. today announced that it will present at the following upcoming investor conferences

• Ultragenyx Reports Third Quarter 2018 Financial Results and Corporate Update

  11/5/2018

  Ultragenyx Pharmaceutical Inc. today reported its financial results and corporate update for the quarter ended September 30, 2018.

• Ultragenyx to Host Conference Call for Third Quarter 2018 Financial Results and Corporate Update

  10/30/2018

  Ultragenyx Pharmaceutical Inc. today announced that it will host a conference call on Monday, November 5, 2018 at 5pm ET to discuss third quarter 2018 financial results and provide a corporate update.

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  Ultragenyx Plunges With Phase III Failure, Company to Discontinue Glut1 DS Development Program

  10/26/2018

  Shares of Ultragenyx are down more than 15 percent this morning after the company announced its Phase III drug candidate UX007 failed to hit the mark as a treatment for patients with glucose transporter type-1 deficiency syndrome.