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Yahoo
a day ago
- Business
- Yahoo
Ultragenyx Pharmaceutical Inc. (RARE) Gets FDA CRL for UX111, Vows Quick Resubmission
We recently compiled a list of Ultragenyx Pharmaceutical Inc. stands first on our list. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), based in Novato, California, focuses on developing therapies for rare and ultra-rare genetic diseases and tops our list for being one of the best growth stocks. The company's portfolio includes commercial products and a strong late-stage pipeline targeting high-need conditions such as Sanfilippo syndrome type A, osteogenesis imperfecta, Angelman syndrome, and glycogen storage diseases. A key recent development was the FDA's Complete Response Letter (CRL) on July 11, 2025, for the company's Biologics License Application for UX111, a gene therapy for Sanfilippo syndrome type A. While the FDA highlighted chemistry, manufacturing, and controls (CMC) issues, it also recognized the robust clinical and biomarker data, affirming the therapy's efficacy. The business intends to resolve the CMC concerns and resubmit the application, expecting a six-month review. Beyond UX111, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is advancing other promising therapies. UX143, targeting osteogenesis imperfecta, is progressing toward final analysis after a Phase 3 study. GTX-102, for Angelman syndrome, recently received the FDA Breakthrough Therapy designation, indicating its potential for accelerated development. The corporation's strategy is notable for its modality-agnostic approach, leveraging gene therapies, monoclonal antibodies, and antisense oligonucleotides. This diversified platform reduces regulatory risk while maximizing impact across several rare disorders. A doctor examining a patient using a vascular disease diagnosis device. Adding to its momentum, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) recently gained Health Canada approval to expand the use of Evkeeza to infants as young as six months with homozygous familial hypercholesterolemia (HoFH), enhancing its commercial reach. While we acknowledge the potential of GOOGL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Medscape
24-07-2025
- Health
- Medscape
Taurine: From Energy Drink Darling to Cancer Suspect?
Taurine enjoys a strong reputation for boosting both physical and mental performance and has long been a staple ingredient in many energy drinks. A 2023 study in Science even suggested that supplementing this amino sulfonic acid — which the body can produce endogenously — could extend life span, at least in mice and monkeys. That research, led by Parminder Singh, PhD, of the Buck Institute in Novato, California, also observed that taurine levels in human blood appear to decline with age. These findings helped fuel a rise in over-the-counter taurine supplement sales. But new evidence suggests that the antiaging effects may be overstated — and that high intake of taurine could even pose risks, including a possible increase in blood cancer susceptibility for some individuals. Poor Fit as Aging Biomarker A more recent Science study casts further doubt on taurine's longevity promise. Maria Emilia Fernandez, PhD, and colleagues at the National Institute on Aging, Baltimore, found that taurine does not serve as a reliable biomarker of biological aging. Unlike the earlier cross-sectional study, their longitudinal research repeatedly measured taurine levels in participants over time. The results showed no consistent age-related decline. In many cases, taurine concentrations increased with age. The researchers concluded that individual variability far outweighed any patterns linked to aging. No Reliable Reference Ranges 'There are no reliable reference values for taurine that could be applied broadly to the general population,' Kristina Norman, MD, head of the Department of Nutrition and Gerontology at the German Institute of Human Nutrition in Potsdam-Rehbrücke, Nuthetal, Germany, told Medscape's German edition . Still, she noted that the study doesn't rule out the possibility that taurine may have beneficial effects in specific contexts — such as for cardiometabolic health. 'That was not the question Fernandez and her colleagues set out to explore,' said Norman, who is also the deputy director of the Research Group in Geriatrics at the Charite Universitätsmedizin Berlin, Berlin, Germany. 'There are a number of interventional studies on taurine supplementation,' Norman added, 'but the results have been inconsistent, which makes interpretation difficult.' Currently, the effects of taurine appear highly dependent on individual factors, including a person's age and underlying health status. As such, Norman advised that taurine supplementation should neither be broadly recommended nor discouraged at this stage. Taurine in the Diet — and in the Data According to the European Food Safety Authority, the maximum safe daily intake of taurine is 100 mg/kg of body weight — about 7 g for a 70-kg adult. European regulations limit taurine in energy drinks to no more than 4 g/L. Taurine also occurs naturally as a breakdown product of the amino acids cysteine and methionine, particularly in meat, fish, seafood, and dairy products. Fernandez's study analyzed data from three human cohorts (973 participants in total) as well as blood samples from rhesus monkeys and mice. The researchers found no evidence of age-related declines in circulating taurine levels among healthy individuals — in fact, levels were often stable or increased with age. Diet, rather than age, showed a stronger correlation with taurine concentrations. Moreover, they found no association between taurine levels and physical parameters such as muscle strength or body weight. 'These findings challenge the prevailing hypothesis that taurine declines with age and contributes to aging,' Norman said. Linked With Leukemia Cell Growth A second study, recently published in Nature , has raised new concerns about potential risks associated with high taurine intake. Researchers led by Sonali Sharma, PhD, from the Department of Biomedical Genetics at the Wilmot Cancer Institute, University of Rochester Medical Center, Rochester, New York, found elevated taurine concentrations in the microenvironment of leukemia cells. They also identified taurine transporters on the cell membranes, which facilitated the uptake of taurine into the cells, where it appeared to support glycolysis. According to Sharma's team, taurine may play a role in both the development and progression of leukemia. 'It's still unclear whether this potential risk applies broadly or only to certain individuals — such as those with a personal or family history of blood cancer,' said Norman. She also noted that it remains uncertain whether orally supplemented taurine even reaches the tumor microenvironment. Nevertheless, she added, the findings open up new therapeutic possibilities — namely, targeting taurine transporters in malignant cells as a novel approach to leukemia treatment. Benefit for Longevity or Performance The Nature study also reinforces the need to re-evaluate taurine's use as a dietary supplement. 'Individuals at increased risk of leukemia should probably avoid taurine supplementation,' Norman advised. More broadly, she noted there is currently no strong evidence to support taurine's use for enhancing physical or cognitive performance or slowing the aging process. 'We simply don't have high-quality data confirming whether taurine works — or at what dose,' Norman said. She also cautioned that taurine's side-effect profile remains poorly understood. 'Taurine could potentially offer benefits in some clinical contexts,' she acknowledged. 'But right now, I would discourage people from taking it on their own 'just in case.''
Yahoo
20-07-2025
- Yahoo
More than 1,600 pictures of child pornography found at Bay Area man's home: PD
(KRON) — A 20-year-old man from Novato was arrested on July 15 for possession of 1,650 pictures of child pornography, according to the Novato Police Department. Detectives with the Novato Police Department searched the home of suspect John Ramirez after they received information that he had allegedly downloaded and viewed child pornography and interacted with minors on the internet. 'NPD Detectives served a search warrant at his residence and located evidence linking him to his crimes,' NPD said. 'Ramirez was not located at the residence and was believed to have fled to Southern California.' Man arrested in Vallejo murder case: PD On July 15, U.S. Marshals arrested Ramirez in Southern California. Ramirez will be extradited to Marin County for possession of child pornography of a minor under 12 years old, and possession of images depicting sexual sadism to a minor, police said. 'This case serves as a serious reminder for parents and guardians to stay actively engaged in their children's online activities,' NPD said. 'Talk to your children about internet safety, monitor their interactions, and report any suspicious behavior to law enforcement immediately.' Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed. Solve the daily Crossword
Wall Street Journal
11-07-2025
- Health
- Wall Street Journal
Step Aside Longevity Bros. It's Time for the Longevity Ladies.
Jennifer Garrison, a 49-years-old neuroscientist, has been studying how aging changes the way the brain communicates with the rest of the body in a lab she runs in Novato, Calif. But on an afternoon in March, she walked across a glitzy Los Angeles stage to the pulsing soundtrack of Kelly Clarkson singing, 'What doesn't kill you makes you stronger' and greeted an audience of mainly high net-worth men gathered to hear about tech and longevity.
Yahoo
04-07-2025
- Business
- Yahoo
QT Imaging to Hold Virtual 2025 Annual Meeting of Stockholders on August 19
NOVATO, Calif., July 04, 2025--(BUSINESS WIRE)--QT Imaging Holdings, Inc. (OTCQB: QTIH), a medical device company engaged in research, development, and commercialization of innovative body imaging systems, today announced that its 2025 Annual Meeting of Stockholders will be held as a virtual meeting on Tuesday, August 19, 2025, at 1:00 p.m. EDT (10:00 a.m. PDT). Stockholders of record at the close of business on July 16, 2025 shall be entitled to vote at the 2025 Annual Meeting. Information about the virtual meeting webcast and instructions for how stockholders can participate in the 2025 Annual Meeting will be included in the definitive proxy statement to be filed with the Securities and Exchange Commission and will be available on the "Investors" section of QT Imaging's website at About QT Imaging Holdings, Inc. QT Imaging Holdings, Inc. is a public (OTCQB: QTIH) medical device company engaged in research, development, and commercialization of innovative body imaging systems using low frequency sound waves. QT Imaging Holdings, Inc. strives to improve global health outcomes. Its strategy is predicated upon the fact that medical imaging is critical to the detection, diagnosis, and treatment of disease and that it should be safe, affordable, accessible, and centered on the patient's experience. For more information on QT Imaging Holdings, Inc., please visit the company's website at Breast Acoustic CT™ is a trademark of an affiliate of QT Imaging Holdings, Inc. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements contain words such as "will," and "expect," or the negative thereof or comparable terminology, and include (without limitation) statements regarding the date of its annual meeting of stockholders. Forward-looking statements involve certain risks and uncertainties, and actual results may differ materially from those discussed in any such statement. These risks include, but are not limited to: research results from the use of the QT Imaging Breast Acoustic CT Scanner, the ability of QT Imaging Holdings to sell and deploy the QT Imaging Breast Acoustic CT Scanner, the ability to extend product offerings into new areas or products, the ability to commercialize technology, unexpected occurrences that deter the full documentation and "bring to market" plan for products, trends and fluctuations in the industry, changes in demand and purchasing volume of customers, unpredictability of suppliers, the ability to attract and retain qualified personnel and the ability to move product sales to production levels. Additional factors that could cause actual results to differ are discussed under the heading "Risk Factors" and in other sections of QT Imaging Holding's filings with the SEC, and in its other current and periodic reports filed or furnished from time to time with the SEC. All forward-looking statements in this press release are made as of the date hereof, based on information available to QT Imaging Holdings as of the date hereof, and QT Imaging Holdings assumes no obligation to update any forward-looking statement. View source version on Contacts For investor and media inquiries, please contact: Stephen Kilmer Head of Investor Stas Budagov Chief Financial Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
