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
A loving mother. An impactful educator. A community leader. All this, wrapped up in an infectious smile. That’s how those touched by the life of Kayla Christian describe her. Christian, a preschool teacher at Grace Early Learning Center in Georgetown, Kentucky, died in November from breast cancer at the age of 33. The beloved teacher
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
Cargo ships load and unload containers at Qingdao Port’s foreign trade container terminal in Qingdao, East China’s Shandong Province, Nov 11, 2021. Yu Fangping | Costfoto | Barcroft Media | Getty Images Ports and companies have been battling the global supply chain crisis since the start of the year. Just as it appeared like the
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
Lung cancer cell. Credit: LRI EM Unit. More than 600 people in England with a form of lung cancer could benefit from the innovative drug osimertinib (Tagrisso) after its approval by the National Institute for Health and Care Excellence (NICE). The drug will now be available to people with early-stage non small cell lung cancer
A novel immunotherapy provided durable disease control in homologous repair proficient (HRP) ovarian cancer, subgroup data from a small randomized trial showed. After 3 years of follow-up, median overall survival (OS) had yet to be reached in patients who received gemogenovatucel-T (Vigil), but appeared to exceed 41 months, as compared with a median OS of
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
What’s better than a smartwatch? A smartwatch with a health tracker and a generous discount, of course! Make sure you don’t miss out on the best deals for Cyber Monday 2021. Wearable health trackers provide the best of both of worlds. So, if you have been bitten by the fitness bug, then today’s your chance to score the best smartwatches
The treadmill may not be your favorite piece of exercise equipment, but you might be swayed after hearing about its effectiveness. If you know how to use it, the machine can work in your favor on multiple levels, including helping you build cardiovascular endurance, strengthen your entire body, and tone your butt and legs. Michael
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
When Mack Simmons was just 17 years old, he selflessly donated his kidney to try and save his sister’s life. But when he was still caring for his ill sister, he wound up becoming sick himself with kidney failure. His pain grew worse every day to the point where he lost his ability to walk
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
A man holds a sign supporting Roe vs. Wade in front of the US Supreme Court Building in Washington, DC on November 30, 2021. Roberto Schmidt | AFP | Getty Images The Supreme Court is about to hear arguments in the most significant abortion case in decades, throwing into doubt the future of Roe v.
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,
Children with poorly-controlled asthma were at much greater risk for COVID-19 hospitalization, a retrospective study from Scotland found. Among roughly 750,000 children ages 5 to 17, those with an asthma-related hospitalization in the 2 years before the pandemic — a marker for uncontrolled asthma — had a more than sixfold higher risk of being hospitalized
KTSDESIGN/SCIENCE PHOTO LIBRARY When supporters donate to our work, their combined generosity might lead to a new discovery that inches us closer to, for example, a new class of life-saving treatments. Or it might help fund a clinical trial, where experimental new drugs are tested. Occasionally – as for those who support our researchers in
Could applying a psychological framework to your work relationships help you to navigate difficult dynamics? Our expert columnist Andy Gill dives into common workplace woes We spend about a third of our lives at work, and wouldn’t it be wonderful if it was a positive experience? Sadly, this is not always the case, and I
“I don’t think I am the right doctor for you.” Before this year, in over 20 years of practice, I had never uttered those words. Now, nearly 2 years into the pandemic, I say it almost daily. I like to think I am a good physician and listener, spending most of the outpatient visit time
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
While the world is on the lookout for possible surges amid the emergence of the omicron variant, early reports appear to suggest that the newer strain does not really cause the severe form of COVID-19 infection. WHO Update On Omicron The World Health Organization has released a new update on the omicron variant, also known as
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
8-year-old Nate Gonzalez may be little, but he has a huge heart. His only wish this Christmas is that other people can have a Merry Christmas and a great life. That’s why he’s working hard to bring smiles to other kids’ faces just in time for the holidays. Last year, he wanted to help kids
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
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
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
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
Train Happy author, podcast host and PT Tally Rye joins Happiful’s podcast to talk about building a positive relationship with exercise and how intuitive movement can help Tally Rye knows all too well how diet culture and ‘fitspo’ mentality can permeate our thinking. As a result, she’s harnessed her own lived experience, training and extensive
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