Ultragenyx Pharmaceutical Inc.

182 articles with Ultragenyx Pharmaceutical Inc.

• 

  CODA Biotherapeutics Launches with $19 Million to Tackle Neuropathic Pain

  9/12/2018

  CODA Biotherapeutics launched in South San Francisco with a $19 million Series A financing.
• 

  REGENXBIO Looks to Take CLN2 Gene Therapy Treatment Into the Clinic

  8/30/2018

  Maryland-based REGENXBIO is expanding its gene therapy pipeline to include a new treatment for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease.
• 

  Debate Over the Orphan Drug Act Heats Up

  8/30/2018

  Recently, Milton Packer, a renowned cardiologist at Baylor Scott & White Health, wrote a criticism of the Orphan Drug Act and the resulting problems caused by the biopharma industry. John LaMattina, formerly president of Pfizer Global Research and Development and a current senior partner at PureT...

• Ultragenyx Reports Second Quarter 2018 Financial Results and Corporate Update

  8/2/2018

  Ultragenyx Pharmaceutical Inc.(NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter ended June 30, 2018.

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

  7/27/2018

  Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, August 2, 2018 at 5pm ET to discuss second quarter 2018 financial results and provide a corporate update.

• Ultragenyx Announces First Patient Dosed in Phase 1/2 study of DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa); FDA Grants Fast Track Designation to Gene Therapy Program

  7/26/2018

  Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the first patient has been dosed in the Phase 1/2 study of DTX401, an adeno-associated virus vector based gene therapy for the treatment of patients with glycogen storage disease type Ia (GSDIa).

• Ultragenyx Announces Positive CHMP Opinion for Mepsevii™ (vestronidase alfa) For the Treatment of Mucopolysaccharidosis VII

  6/29/2018

  ltragenyx Pharmaceutical Inc.(NASDAQ:RARE) today announced that the Committee for Medicinal Products for Human Use(CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion recommending the marketing authorization under exceptional circumstances of Mepsevii™ (vestronidase alfa) for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

• Aimmune Therapeutics Appoints Dr. Jayson Dallas as CEO

  6/11/2018

  Aimmune Therapeutics, Inc. today announced the appointment of Jayson Dallas, M.D., as President and Chief Executive Officer.

• PANTHERx® Specialty Pharmacy Selected by Ultragenyx to Distribute Crysvita

  6/7/2018

  Crysvita™ is the very first FDA-approved therapy for children and adults with x-linked hypophosphatemia

• Ultragenyx to Present at Upcoming Investor Conferences .

  6/4/2018

  Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, will present at the following investor conferences.

• Rentschler Fill Solutions and Ultragenyx Start Fill & Finish Collaboration for the US Commercial Supply of Drug Product Mepsevii

  5/30/2018

  Rentschler Fill Solutions GmbH announced a collaboration with Ultragenyx Pharmaceutical, Inc. for the drug product production of the injectable MepseviiTM.

• Ultragenyx and Kyowa Kirin Announce Publication of Phase 2 Study Results Demonstrating that Crysvita® (burosumab) Improved Outcomes in Children with X-linked Hypophosphatemia in the New England Journal of Medicine

  5/23/2018

  Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), Kyowa Hakko Kirin Co. Ltd, and Kyowa Kirin International PLC today announced that the results of a pediatric Phase 2 clinical trial of Crysvita® (burosumab) for the treatment of X-linked hypophosphatemia (XLH) in children aged 5 to 12 years were published online by the New England Journal of Medicine (NEJM).

• 

  Who are the Top 3 Biotech Takeover Targets?

  5/22/2018

  Most investors thought 2018 would be a big year for mergers and acquisitions in the biopharma industry because of changes to the tax law, and so far they’ve been right.

• Ultragenyx and Kyowa Kirin Announce Topline Phase 3 Study Results Demonstrating Superiority of Crysvita® (burosumab) Treatment to Oral Phosphate and Active Vitamin D in Children with X-Linked Hypophosphatemia (XLH)

  5/17/2018

  Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co. Ltd(Kyowa Hakko Kirin), and Kyowa Kirin International PLC today announced that the Phase 3 study of Crysvita® (burosumab) met its primary endpoint.

• Ultragenyx Reports First Quarter 2018 Financial Results and Corporate Update

  5/7/2018

  Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter ended March 31, 2018.

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

  5/1/2018

  ltragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Monday, May 7, 2018 at 5pm ET to discuss first quarter 2018 financial results and provide a corporate update.

• Ultragenyx and Kyowa Kirin Announce Crysvita® (burosumab-twza) Now Launched in the U.S. for the Treatment of X–linked Hypophosphatemia (XLH) in Children and Adults

  4/30/2018

  Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co. Ltd, and Kyowa Kirin International PLC today announced that Crysvita® (burosumab-twza) entered the commercial supply chain in the United States and generated its first sales to specialty pharmacies on April 27, 2018.

• Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia

  4/23/2018

  Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration(FDA) has cleared the Investigational New Drug (IND) application for DTX401, an adeno-associated virus vector based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa).

• Ultragenyx and Kyowa Kirin Announce FDA Approval of Crysvita® (burosumab-twza) for the Treatment of Children and Adults with X–Linked Hypophosphatemia (XLH)

  4/17/2018

  ltragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co. Ltd(Kyowa Hakko Kirin), and Kyowa Kirin International PLC (Kyowa Kirin International) today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab-twza) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older.

• Ultragenyx to Present at Upcoming Investor Conferences . .

  3/8/2018

  Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, will present at the following investor conferences.