YENOM NOTICIAS
Noticias financieras organizadas para conectar titulares, símbolos, fuente, fecha y sentimiento con tu análisis y operativa.
Si escribes ticker, YENOM prioriza ese instrumento. Si lo dejas vacío puedes explorar el feed general o por tema.
Ultragenyx Pharmaceutical RARE has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million. The transaction is expected to provide the rare-disease drugmaker with a meaningful inf…
Ultragenyx Pharmaceutical (RARE) is back in focus after the European Medicines Agency validated its Marketing Authorisation Application for rebisufligene etisparvovec, a gene therapy targeting ultra-rare Sanfilippo syndrome Type A. Against…
Ultragenyx Pharmaceutical Inc. NOVATO, Calif., Oct. 07, 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…
On 2 October 2026, Ultragenyx announced that the European Medicines Agency had validated its Marketing Authorisation Application for rebisufligene etisparvovec, an investigational AAV9 gene therapy for ultra-rare Sanfilippo syndrome Type A…
Ultragenyx Pharmaceutical RARE announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for the investigational gene therapy rebisufligene etisparvovec (also known as UX111) to treat mu…
Ultragenyx Pharmaceutical Inc. BASEL, Switzerland, Oct. 02, 2026 (GLOBE NEWSWIRE) -- Today Ultragenyx announced that the European Medicines Agency (EMA) has validated the Company's Marketing Authorisation Application (MAA) for its investig…
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) just added another commercial product to its portfolio. The FDA on September 17 approved Fayuvi for the treatment of neurologic manifestations of Sanfilippo syndrome Type A in pediatric patients…
In September 2026, Ultragenyx Pharmaceutical received full U.S. FDA approval for FAYUVI (UX111), the first-ever treatment for pediatric patients with Sanfilippo syndrome Type A, along with a Priority Review Voucher. This approval not only…
Shares of Ultragenyx Pharmaceutical RARE jumped 12.6% on Thursday after the FDA granted standard full approval to Fayuvi (rebisufligene etisparvovec-hopf), also known as UX111. The one-time gene therapy is approved for treating the neurolo…
COLUMBUS, Ohio, Sept. 18, 2026 /PRNewswire/ -- Andelyn Biosciences ("Andelyn"), a leading, patient-focused cell and gene therapy Contract Development and Manufacturing Organization ("CDMO"), today announced it is now manufacturing FAYUVI™…
Abeona Therapeutics Inc. - FDA approval represents a monumental milestone for patients with MPS IIIA and validates Abeona's foundational R&D and clinical development efforts - - Ultragenyx successfully brought a life-changing therapy acros…
Ultragenyx (RARE) shares ended the last trading session 12.6% higher at $14.5. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 50.9% loss over th…
Ultragenyx Pharmaceuticals won approval for its newest gene therapy on Thursday, helping shares partially reverse a catastrophic dive. Continue Reading
The biopharmaceutical company received FDA approval for a first-of-its-kind treatment for a rare neurodevelopmental disease. Continue Reading
Ultragenyx Pharmaceutical Inc. FAYUVI 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 Typ…
[Paper with words Sanfilippo syndrome and glasses.] * The US FDA has approved Ultragenyx Pharmaceutical's (RARE [https://seekingalpha.com/symbol/RARE]) gene therapy Fayuvi (rebisufligene etisparvovec) for pediatric treatment of Sanfilippo…
In early September 2026, Ultragenyx Pharmaceutical announced that its Phase 3 Aspire trial of apazunersen (GTX-102) in Angelman syndrome failed to meet its primary and key secondary cognitive and functional endpoints, prompting a review of…
Ultragenyx Pharmaceutical stock is under pressure after a multi year slide in returns, yet current market multiples still screen as expensive and the broader valuation checks send a mixed message. With fresh clinical setbacks alongside som…
Ultragenyx stock crashed Thursday, losing almost half its value, after its experimental Angelman treatment failed in final-phase testing. Continue Reading
Nvidia stock rises on a deal for it to acquire Hugging Face while Tesla sadvances ahead of an important Cybercab event. Continue Reading
Ultragenyx Pharmaceuticals looks to cut costs following the trial failure, while attention remains squared on its burgeoning commercial business. Continue Reading
[An arrow pointing downwards on top of a pile of US dollar bills.] Ultragenyx Pharmaceutical (RARE [https://seekingalpha.com/symbol/RARE]) plunged more than 46% to an all-time low on Thursday amid multiple downgrades spurred by a late-stag…
It has been about a month since the last earnings report for Ultragenyx (RARE). Shares have added about 6.4% in that time frame, outperforming the S&P 500. But investors have to be wondering, will the recent positive trend continue leading…
Ultragenyx Pharmaceutical RARE stock is down 46% in premarket trading today after the company reported that the late-stage study evaluating its investigational antisense oligonucleotide candidate, apazunersen (GTX-102), for Angelman syndro…
Nvidia stock rises on a deal for it to acquire Hugging Face whiule Tesla stock advances ahead of an important Cybercab event. Continue Reading
The most talked about and market moving research calls around Wall Street are now in one place. Here are today's research calls that investors need to know, as compiled by The Fly. Top 5 Upgrades: Macquarie upgraded Broadcom (AVGO) to Outp…
Quick Read Broadcom, Deere, and Dell all earned Outperform upgrades Thursday, with analyst price targets set at $490, $813, and $625 respectively. Moderna was cut to Sell with an $81 target, while Ultragenyx's price target was halved to $1…
Check out the companies making the biggest moves premarket: Snowflake — Shares surged 24% after the company's second-quarter results topped analyst expectations. For the quarter, Snowflake posted adjusted earnings of 62 cents per share on…
Pipette dropping liquid into a test tube ©Adobe Stock Images Ultragenyx (NASDAQ:RARE) shares fell 44.7% to $14.67 in premarket trading after the company disclosed that its pivotal Phase 3 Aspire study evaluating apazunersen (GTX-102) in An…
Snowflake shares soared Thursday after the data cloud company reported better-than-expected earnings on strong AI demand. Credit: Michael M. Santiago/Getty Images Stocks finished sharply higher Thursday as Treasury yields declined followin…
Investing.com -- U.S. equity futures were mixed early Thursday, a day after major averages snapped a three-day losing streak driven by rising Treasury yields amid renewed U.S.-Iran hostilities. S&P 500 futures were up 0.1%, Dow futures ros…
[Woman scientist in lab look at science microscope medical test and research biology chemistry. Females technician laboratory analyzing scientific pharmacy genetic research. Chemistry Medical test lab] howtogoto * Ultragenyx Pharmaceutical…
Ultragenyx Pharmaceutical Inc. Phase 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.,…
Ultragenyx Pharmaceutical (RARE) recently received accelerated approval from the U.S. Food and Drug Administration for GENGLYCOS, a gene therapy for glycogen storage disease type Ia. This milestone expands its portfolio of treatments for r…
Ultragenyx Pharmaceutical Inc. At 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…
Rare diseases have moved steadily from being an overlooked corner of medicine to an important frontier for drug innovation. The FDA estimates that more than 7,000 rare diseases affect more than 30 million Americans, while most of these con…