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 PRESS RELEASE

Ultragenyx to Host Conference Call for Second Quarter 2025 Financial R...

Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update NOVATO, Calif., July 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, August 5, 2025, to discuss its financial results and corporate update for the quarter ending June 30, 2025. The live and replayed webcast of the call will be available thro...

 PRESS RELEASE

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(...

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., July 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 45,292 restricted stock units of the company’s common stock to 29 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan...

 PRESS RELEASE

Health Canada Extends the Approval of Evkeeza® (evinacumab) to Childre...

Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH) First and only medicine approved by Health Canada for patients as young as 6-months of age with HoFH, an ultrarare, inherited form of high cholesterol TORONTO, July 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of ...

 PRESS RELEASE

Santé Canada étend l’approbation d’Evkeeza® (évinacumab) chez les enfa...

Santé Canada étend l’approbation d’Evkeeza® (évinacumab) chez les enfants âgés d’à peine 6 mois atteints d’hypercholestérolémie familiale homozygote (HFHo) Premier et seul médicament approuvé par Santé Canada chez les patients âgés d’à peine 6 mois atteints d’HFHo, une forme héréditaire très rare d’hypercholestérolémie TORONTO, 14 juill. 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ : RARE), une société biopharmaceutique spécialisée dans la mise au point et la commercialisation de nouveaux traitements contre des maladies génétiques rares et très rares, a annoncé aujour...

 PRESS RELEASE

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Ge...

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable FDA clinical reviews acknowledged that the clinical data are robust and biomarker data are supportive NOVATO, Calif., July 11, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License...

Dylan Shindler ... (+2)
  • Dylan Shindler
  • Laura Chico
 PRESS RELEASE

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for ...

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis Data from Orbit and Cosmic studies expected around the end of the year NOVATO, Calif., July 09, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO), today announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis consistent with the orig...

Wedbush Research
  • Wedbush Research
UBI UBISOFT ENTERTAINMENT SA
XNCR XENCOR INC.
XENE XENON PHARMACEUTICALS INC
VYGR VOYAGER THERAPEUTICS INC.
TTWO TAKE-TWO INTERACTIVE SOFTWARE INC.
TSLA TESLA INC
RARE ULTRAGENYX PHARMACEUTICAL INC.
PRGS PROGRESS SOFTWARE CORPORATION
NVDA NVIDIA CORPORATION
NVCR NOVOCURE LTD.
NBIX NEUROCRINE BIOSCIENCES INC.
KURA KURA ONCOLOGY INC.
GOOGL ALPHABET INC. CLASS A
GME GAMESTOP CORP. CLASS A
GERN GERON CORP.
FB FACEBOOK INC. CLASS A
FATE FATE THERAPEUTICS INC
CTMX CYTOMX THERAPEUTICS INC.
BPMC BLUEPRINT MEDICINES CORP.
BMRN BIOMARIN PHARMACEUTICAL INC.
BIIB BIOGEN INC.
ASND ASCENDIS PHARMA A/S SPONSORED ADR
ARDX ARDELYX INC
FRX_CN FENNEC PHARMACEUTICALS
7974 NINTENDO CO. LTD.
EA ELECTRONIC ARTS INC.
PTGX PROTAGONIST THERAPEUTICS
ANAB ANAPTYSBIO INC.
ARGX ARGENX ADS
MRSN MERSANA THERAPEUTICS
ISR ISORAY
AMZN AMAZON.COM INC.
MSFT MICROSOFT CORPORATION
APLS APELLIS PHARMACEUTICALS
DNLI DENALI THERAPEUTICS INC
RCUS ARCUS BIOSCIENCES
SRRK SCHOLAR ROCK HOLDING CORPORATION
KNSA KINIKSA PHARMACEUTICALS
REPL REPLIMUNE GROUP
ARVN ARVINAS HOLDING
YMAB Y-MABS THERAPEUTICS
STRO SUTRO BIOPHARMA
STOK STOKE THERAPEUTICS
IGMS IGM BIOSCIENCES
SWTX SPRINGWORKS THERAPEUTICS
IDYA IDEAYA BIOSCIENCES
PASG PASSAGE BIO
BDTX BLACK DIAMOND THERAPEUTICS
ACET ADICET BIO INC
KROS KEROS THERAPEUTICS
ITOS ITEOS THERAPEUTICS
ZNTL ZENTALIS PHARMACEUTICALS
LRMR LARIMAR THERAPEUTICS INC
PRAX PRAXIS PRECISION MEDICINES
U UNITY SOFTWARE
TVTX TRAVERE THERAPEUTICS INC
VRDN VIRIDIAN THERAPEUTICS INC
PLTK PLAYTIKA HOLDING
BUR BURFORD CAPITAL
RBLX ROBLOX
DAWN DAY ONE BIOPHARMACEUTICALS INC
ELEV ELEVATION ONCOLOGY
APP APPLOVIN CORP
ATXS ASTRIA THERAPEUTICS INC
GMTX GEMINI THERAPEUTICS INC
VIGL VIGIL NEUROSCIENCE INC
TYRA TYRA BIOSCIENCES INC
EWTX EDGEWISE THERAPEUTICS INC
DDI DOUBLEDOWN INTERACTIVE CO LTD
APGE APOGEE THERAPEUTICS INC
SKLZ SKILLZ INC
David Nierengarten ... (+8)
  • David Nierengarten
  • Dennis Pak
  • Dylan Shindler
  • Laura Chico
  • Martin Fan
  • Ritika Das
  • Robert Driscoll
  • Yun Zhong
 PRESS RELEASE

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in An...

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment for Angelman syndrome. "FDA Breakthrough Therapy Designation underscores...

 PRESS RELEASE

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(...

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) NOVATO, Calif., June 20, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 48,731 restricted stock units of the company’s common stock to 26 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan...

 PRESS RELEASE

Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthca...

Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference NOVATO, Calif., June 03, 2025 (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 that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025, at 10:00 a.m. ET. The live and archived webcast of the panel ...

Wedbush Research
  • Wedbush Research
UBI UBISOFT ENTERTAINMENT SA
XNCR XENCOR INC.
XENE XENON PHARMACEUTICALS INC
VYGR VOYAGER THERAPEUTICS INC.
TTWO TAKE-TWO INTERACTIVE SOFTWARE INC.
RARE ULTRAGENYX PHARMACEUTICAL INC.
NVCR NOVOCURE LTD.
NBIX NEUROCRINE BIOSCIENCES INC.
KURA KURA ONCOLOGY INC.
GME GAMESTOP CORP. CLASS A
GERN GERON CORP.
FATE FATE THERAPEUTICS INC
CTMX CYTOMX THERAPEUTICS INC.
BPMC BLUEPRINT MEDICINES CORP.
BMRN BIOMARIN PHARMACEUTICAL INC.
BIIB BIOGEN INC.
ASND ASCENDIS PHARMA A/S SPONSORED ADR
ARDX ARDELYX INC
CDR CD PROJEKT S.A.
FRX_CN FENNEC PHARMACEUTICALS
7974 NINTENDO CO. LTD.
BLDR BUILDERS FIRSTSOURCE INC.
EA ELECTRONIC ARTS INC.
ANAB ANAPTYSBIO INC.
ARGX ARGENX ADS
MRSN MERSANA THERAPEUTICS
ISR ISORAY
AAPL APPLE INC.
MSFT MICROSOFT CORPORATION
APLS APELLIS PHARMACEUTICALS
DNLI DENALI THERAPEUTICS INC
RCUS ARCUS BIOSCIENCES
SRRK SCHOLAR ROCK HOLDING CORPORATION
KNSA KINIKSA PHARMACEUTICALS
REPL REPLIMUNE GROUP
ARVN ARVINAS HOLDING
YMAB Y-MABS THERAPEUTICS
STRO SUTRO BIOPHARMA
PINS PINTEREST INC. CLASS A
STOK STOKE THERAPEUTICS
IGMS IGM BIOSCIENCES
SWTX SPRINGWORKS THERAPEUTICS
APRE APREA THERAPEUTICS
IDYA IDEAYA BIOSCIENCES
RVMD REVOLUTION MEDICINES
PASG PASSAGE BIO
BDTX BLACK DIAMOND THERAPEUTICS
ACET ADICET BIO INC
KROS KEROS THERAPEUTICS
ITOS ITEOS THERAPEUTICS
ZNTL ZENTALIS PHARMACEUTICALS
LRMR LARIMAR THERAPEUTICS INC
PRAX PRAXIS PRECISION MEDICINES
U UNITY SOFTWARE
TVTX TRAVERE THERAPEUTICS INC
UFPI UFP INDUSTRIES INC.
VRDN VIRIDIAN THERAPEUTICS INC
IMNM IMMUNOME
PLTK PLAYTIKA HOLDING
NUVB NUVATION BIO INC (A)
RBLX ROBLOX
CGEM CULLINAN ONCOLOGY INC
DAWN DAY ONE BIOPHARMACEUTICALS INC
ELEV ELEVATION ONCOLOGY
TCRX TSCAN THERAPEUTICS INC
APP APPLOVIN CORP
ATXS ASTRIA THERAPEUTICS INC
GMTX GEMINI THERAPEUTICS INC
VIGL VIGIL NEUROSCIENCE INC
TYRA TYRA BIOSCIENCES INC
EWTX EDGEWISE THERAPEUTICS INC
NUVL NUVALENT INC
ARTV ARTIVA BIOTHERAPEUTICS INC
DDI DOUBLEDOWN INTERACTIVE CO LTD
APGE APOGEE THERAPEUTICS INC
SKLZ SKILLZ INC
EMBRAC B EMBRACER GROUP AB
BCAX BICARA THERAPEUTICS INC.
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