When the human genome was fully sequenced in 2003, the prospects of genetic medicine ushered in a new era of human health. Today, scientists and drug developers have a better understanding of the genetic drivers of disease than ever before. As such, genomics stocks present a new opportunity for investors to buy into the potential future of medicine, as nearly all modern therapies rely on understanding patient genetics to be as efficient as possible. Moreover, genomics companies differ from tradi

Ultragenyx (RARE) reports encouraging second-quarter 2024 results, beating both earnings and revenue estimates driven by the year-over-year increase of Crysvita and Dojolvi sales.

RARE) on Thursday reported a loss of $131.6 million in its second quarter. On a per-share basis, the Novato, California-based company said it had a loss of $1.52. The results exceeded Wall Street expectations.

Second quarter total revenue of $147 million, Crysvita® revenue of $114 million and Dojolvi® revenue of $19 million Increased 2024 expected total revenue guidance to $530 million to $550 million NOVATO, Calif., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended June

NOVATO, Calif., July 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, August 1, 2024, to discuss its financial results and corporate update for the quarter ending June 30, 2024. The live and replayed webcast of the call will be available through th

Presentations will include an encore of the positive interim Phase 1/2 results presented in April at the 76th Annual American Academy of Neurology (AAN) MeetingNOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndr

Ionis Pharmaceuticals stock surged Monday after the company unveiled promising results for a drug that treats a rare genetic condition.

NOVATO, Calif., July 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 19,875 restricted stock units of the company’s common stock to 14 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 Emplo

Ultragenyx (RARE) reaches alignment with the FDA regarding the pivotal phase III study design and endpoints for GTX-102 for Angelman syndrome, which is scheduled to be initiated later this year.

Alignment with FDA on Phase 3 study primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI) Phase 3 study on track to initiate by the end of this year NOVATO, Calif., July 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the successful completion of an end-of-Phase 2 (EoP2) meeting with the U.S. Food and Drug Administration (FDA), supporting its Phase 3 study plans for GTX-102, an antisense oligonucleotide