$RARE News

Last published 11h ago

11h agoRAREUltragenyx to Participate at Investor Conferences in LondonGlobeNewswireNOVATO, Calif., Nov. 11, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced participation in upcoming investor conferences.
5d agoRAREUltragenyx Announces the Marketing Authorisation Application (MAA) for its Investigational Gene Therapy for MPS IIIA (Sanfilippo syndrome Type A) is Under Review with the UK Medicines and Healthcare Products Regulatory Agency (MHRA)GlobeNewswireBASEL, Switzerland, Nov. 06, 2026 (GLOBE NEWSWIRE) -- Ultragenyx (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases,
5d agoRAREUltragenyx Announces the Marketing Authorisation Application (MAA) for its Investigational Gene Therapy for MPS IIIA (Sanfilippo syndrome Type A) is Under Review with the UK Medicines and Healthcare Products Regulatory Agency (MHRA)GlobeNewswireBASEL, Switzerland, Nov. 06, 2026 (GLOBE NEWSWIRE) -- Ultragenyx (NASDAQ:RARE), a biopharmaceutical company focused on the development and...
1w agoRAREUltragenyx Pharmaceutical Inc. - EX-99.1Earnings ReleasesForm 8-K - Current report - Item 2.01 Item 2.02 Item 2.05 Item 9.01
1w agoRAREUltragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027GlobeNewswireThird quarter total revenue of $173 million, CRYSVITA® revenue of $109 million and DOJOLVI® revenue of $30 million GENGLYCOS™ and FAYUVI™ BLAs approved by FDA in the third quarter Two Priority Review Vouchers
2w agoRAREUltragenyx to Host Conference Call for Third Quarter 2026 Financial Results and Corporate UpdateGlobeNewswireNOVATO, Calif., Oct. 27, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and
4w agoRAREUltragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $203 MillionGlobeNewswireNOVATO, Calif., Oct. 14, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher
1mo agoRAREULTRAGENYX CONFERENCE CALL - ULTRAGENYX PHARMACEUTICAL INCEarnings Call TranscriptsGood afternoon and welcome to the Ultragenyx Pharmaceutical Conference Call to discuss the U.S. Food and Drug Administration approval of the FeUV for the treatment of the MPSIIIIA, also known as Sanfilippo Syndrome Type
1mo agoRAREULTRAGENYX CONFERENCE CALL - ULTRAGENYX PHARMACEUTICAL INCEarnings Call TranscriptsGood afternoon and welcome to the Ultragenyx Pharmaceutical Conference Call to discuss the U.S. Food and Drug Administration approval of the FeUV for the treatment of the MPSIIIIA, also known as Sanfilippo Syndrome Type
1mo agoRAREUltragenyx Announces Approval of FAYUVI™ Gene Therapy, the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA)GlobeNewswireFAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A
1mo agoJBSS MSM RAREFDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type AFDA Press ReleasesThe U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A., MPS IIIA is a rare inherited disease that progressively damages the brain and nervous system, causing children to lose cognitive, language and other developmental abiliti ...
2mo agoRAREUltragenyx Announces Phase 3 Aspire results in Angelman SyndromeGlobeNewswirePhase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI) NOVATO, Calif., Sept.
2mo agoRAREUltragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic DiseaseGlobeNewswireAt Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50%
2mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., Aug. 25, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
2mo agoRAREULTRAGENYX CORPORATE UPDATE - ULTRAGENYX PHARMACEUTICAL INCEarnings Call TranscriptsGood afternoon and welcome to the Ultragenyx Pharmaceutical Conference Call to discuss the U.S. Food and Drug Administration approval of GenGlycos, known as DTX401, for the treatment of glycogen storage disease type 1a o
2mo agoRAREUltragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)GlobeNewswireGENGLYCOS is the first gene therapy approval, and fifth FDA approval overall, for the company Approval provides a long-awaited first-ever option to reduce the burden of care associated with GSDIa Ultragenyx received a
2mo agoMSM RAREFDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type IaFDA Press ReleasesThe U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) to reduce daily cornstarch intake as an adjunct to nutritional management in adults and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa), a rare, inherited genetic disorder caused by a deficiency of the enzyme glucose-6-phosphatase, whi ...
3mo agoRAREULTRAGENYX SECOND QUARTER 2026 EARNINGS CALL - ULTRAGENYX PHARMACEUTICAL INCEarnings Call TranscriptsGood afternoon, and welcome to the Ultragenyx Second Quarter 2026 Financial Results Conference Call. At this time, all participants are in a listen-only mode. At the end of the prepared remarks, you will have an opportun
3mo agoRAREUltragenyx Second Quarter 2026 Earnings Call - ULTRAGENYX PHARMACEUTICAL INCEarnings Call TranscriptsGood afternoon, and welcome to the Ultragenyx Second Quarter 2026 Financial Results Conference Call. At this time, all participants are in a listen-only mode. At the end of the prepared remarks, you will have an opportun
3mo agoRAREUltragenyx Reports Second Quarter 2026 Financial Results and Corporate UpdateGlobeNewswireSecond quarter total revenue of $214 million, Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and
3mo agoRAREUltragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate UpdateGlobeNewswireNOVATO, Calif., July 28, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and
3mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., July 24, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
4mo agoRAREUltragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global ImpactGlobeNewswireNOVATO, Calif., July 08, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) released its 2025 Impact Report, detailing the company's work on behalf of patients and families living with rare and
4mo agoCLDI LEGN RARECalidi Biotherapeutics Appoints Former Genentech/Roche and Celgene Executive Corsee Sanders, Ph.D., to Board of DirectorsGlobeNewswireSAN DIEGO, June 23, 2026 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics, Inc. (NYSE:CLDI) ("Calidi" or "the Company"), a biotechnology company pioneering the development of targeted genetic medicines,
4mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., June 19, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
5mo agoRAREKuehn Law Encourages Investors of Ultragenyx Pharmaceutical Inc. to Contact Law FirmPR NewswireKuehn Law, PLLC, a shareholder litigation law firm, is investigating whether certain officers and directors of Ultragenyx Pharmaceutical Inc....
5mo agoRAREUltragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare ConferenceGlobeNewswireNOVATO, Calif., June 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and
5mo agoRAREKuehn Law Encourages Investors of Ultragenyx Pharmaceutical Inc. to Contact Law FirmGlobeNewswireNEW YORK, June 02, 2026 (GLOBE NEWSWIRE) -- Kuehn Law, PLLC, a shareholder litigation law firm, is investigating whether certain officers and directors of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) breached their
5mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., May 22, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
6mo agoRARERare disease families find roadmap to drug development at bootcampsCNBCUltragenyx’s Rare Bootcamp gives families a roadmap to help drive research and develop new treatments for rare diseases.
6mo agoRAREUltragenyx to Participate at Bank of America's 2026 Healthcare ConferenceGlobeNewswireNOVATO, Calif., May 06, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and
6mo agoRAREUltragenyx Reports First Quarter 2026 Financial Results and Corporate UpdateGlobeNewswireFirst quarter total revenue of $136 million, Crysvita® revenue of $93 million and Dojolvi® revenue of $18 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and
6mo agoRAREUltragenyx to Host Conference Call for First Quarter 2026 Financial Results and Corporate UpdateGlobeNewswireNOVATO, Calif., April 29, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and
6mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., April 24, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
7mo agoRAREUltragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)GlobeNewswireIf approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration and early death
7mo agoRAREUltragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE MyopathyGlobeNewswireNOVATO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND)
8mo agoRAREUltragenyx Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) DeficiencyGlobeNewswireStatistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeks Clinically important changes observed in patient global impression scale for overall OTC symptoms, OTC
8mo agoRAREUltragenyx to Participate at Investor Conferences in MarchGlobeNewswireNOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and
8mo agoRAREUltragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)GlobeNewswirePDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.
8mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., Feb. 20, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
9mo agoRAREUltragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate UpdateGlobeNewswire2025 total revenue of $673 million,Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million 2026 total revenue from current products expected to be between $730 million to $760 million
9mo agoRAREInvestors in Ultragenyx Pharmaceutical Inc. Should Contact The Gross Law Firm Before April 6, 2026 to Discuss Your Rights - RAREPR NewswireThe Gross Law Firm issues the following notice to shareholders of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE). Shareholders who purchased shares of ...
9mo agoRAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2025 Financial Results and Corporate UpdateGlobeNewswireNOVATO, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and
9mo agoRAREUltragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA)GlobeNewswireData represents up to 8.5 years of follow-up and are consistent across age, dose, and genotype BLA resubmitted to U.S. FDA in January 2026; Company expects up to six-month review period per FDA guidelines NOVATO,
9mo agoRAREUltragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDAGlobeNewswireCompany expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be presented at
9mo agoRAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswireNOVATO, Calif., Jan. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare
10mo agoRAREUltragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare ConferenceGlobeNewswirePreliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investments of approximately $735 million as of December 31, 2025 Anticipated 2026 catalysts include two
10mo agoRAREUltragenyx to Present at the 44th Annual J.P. Morgan Healthcare ConferenceGlobeNewswireNOVATO, Calif., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and
10mo agoRAREUltragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)GlobeNewswireNOVATO, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food
10mo agoRAREUltragenyx Announces Phase 3 Orbit and Cosmic Results for Setrusumab (UX143) in Osteogenesis ImperfectaGlobeNewswireNeither study achieved its primary endpoint of reduction in annualized clinical fracture rate compared to placebo (Orbit) or bisphosphonates (Cosmic) Both studies achieved the secondary endpoint of improvements in bone