This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional dystrophin proteins, as described in the peer-reviewed journal Nucleic Acid Therapeutics. Golodirsen was tested in ambulatory patients with exon 53 skip amenable DMD. They
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Many countries have implemented various nonpharmaceutical interventions (NPIs) such as social distancing mandates and lockdowns in response to the coronavirus disease 19 (COVID-19) pandemic. Although these measures reduced stress on the healthcare system in the absence of vaccines, they were associated with major socioeconomic costs. This led to a revival of the debate on whether
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Breast cancer is the most common cancer with the highest mortality rate. Swift detection and diagnosis diminish the impact of the disease. However, classifying breast cancer using histopathology images -; tissues and cells examined under a microscope -; is a challenging task because of bias in the data and the unavailability of annotated data in
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Men with localized prostate cancer who opt for radical prostatectomy are more likely to regret their decision than those who opt for radiotherapy or active surveillance, according to a survey study of 2072 patients. At 5 years, 13% of men surveyed experienced treatment-related regret, which varied by treatment type — 16% (183) of surgery patients
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In pediatric and young adult patients with acute lymphoblastic leukemia (ALL) treated with tisagenlecleucel (Kymriah), DNA sequencing-based detection of residual disease between three and 12 months accurately identified all patients who would eventually relapse, while other methods were less predictive. Journal in Which the Study was Published: Blood Cancer Discovery, a journal of the American
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In the first MRI-based study to investigate pre-natal alcohol exposure, researchers found significant changes in the brain structure of fetuses exposed to alcohol compared to healthy controls. Results of the study are being presented today at the annual meeting of the Radiological Society of North America (RSNA). Fetal alcohol syndrome is a worldwide problem in
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The severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) outbreak in Wuhan, China, was first reported on December 31st, 2019. These initial coronavirus disease 2019 (COVID-19) cases caused by SARS-CoV-2 were linked to an animal wet market. Similarities between SARS-CoV-2 and other coronaviruses suggest that bats may be natural hosts and potential reservoirs for this virus,
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Editor’s note: Find the latest COVID-19 news and guidance in Medscape’s Coronavirus Resource Center. A US Food and Drug Administration (FDA) advisory panel narrowly voted 13 to 10 today to recommend emergency use authorization for the antiviral pill molnupiravir from Merck and Ridgeback Biotherapeutics to treat COVID-19. According to the panel of experts, the drug may
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As the COVID-19 pandemic nears the two-year mark, Northwestern Medicine scientists continue to tackle every facet of the disease, from investigating coronavirus vaccines’ potential for providing immunity against similar coronaviruses to developing novel rapid antigen-based tests and examining disparities in COVID-19 case and mortality rates in Chicago. Coronavirus vaccines may provide cross-protective immunity Northwestern Medicine
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In mice with high-risk neuroblastoma, tumors disappeared in response to a new combination treatment with precision medicines, a recent study from University of Gothenburg researchers shows. This is a vital step toward a potentially curative treatment for a form of cancer affecting young children that is currently difficult to treat. The study, published in the
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Elizabeth Holmes, founder of Theranos Inc., arrives at federal court in San Jose, California, U.S., on Tuesday, Nov. 30, 2021. David Paul Morris | Bloomberg | Getty Images SAN JOSE, CALIF. — In her first day facing questions from the prosecution, Theranos founder Elizabeth Holmes admitted that she tried to get News Corp’s Rupert Murdoch
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A new study investigates the potential effects of low-quality sleep on brain development in early adolescence. It’s no secret that sleep is crucial for optimal health and well-being.  Many different functions occur during periods of sleep, and the body takes that time to repair, rest, and rejuvenate itself.1  Research shows that sleep may play an important role
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There are few treatment options for children with multiple sclerosis-;a condition in which the immune system attacks the protective covering of nerves in the brain and spinal cord-;and most therapies for the disease have not been tested in children. An international team of investigators, including researchers at Massachusetts General Hospital (MGH), has conducted a phase
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