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Clinical trial shows newborns with spinal muscular atrophy can start treatment at birth

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  • 2025-08-14 20:17 event
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Clinical trial shows newborns with spinal muscular atrophy can start treatment at birth
Spinal muscular atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness, which, when untreated, prevents infants with the most severe form from gaining motor development—never gaining the ability to sit—and typically leads to death before 2 years of age.

171. Cancer drug eliminates aggressive cancers in Phase I trial

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Over the past 20 years, a class of cancer drugs called CD40 agonist antibodies have shown great promise—and induced great disappointment.

172. Understanding autism: Early signs, misleading labels and why support matters

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Autism spectrum disorder is a complex neurodevelopmental condition that affects how individuals communicate, behave and interact with others. Despite increasing awareness, misconceptions about autism persist—from when it can be detected to whether it can be "cured."

173. Longevity is going to change almost all aspects of our lives, says psychologist

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As 100-year lives become increasingly common, Stanford psychologist Laura Carstensen is working to understand the implications—and to ensure we can make the most of our extra years. The following are the researchers' own words, edited and condensed for clarity.

174. High triglycerides drive life-threatening aortic aneurysms, study in mice finds

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High levels of triglycerides, the most common type of fat in the body and the foods we eat, directly cause abdominal aortic aneurysms, according to a study in mouse models led by Michigan Medicine.

175. Minimally invasive procedure relieves painful symptoms of knee osteoarthritis

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A procedure performed under mild sedation in less than two hours by an interventional radiologist relieves chronic knee pain caused by osteoarthritis, an NYU Langone Health study shows.

176. Basophil Activation Test outperforms current tests for diagnosing milk allergy in young children

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A study led by researchers from King's College London and Guy's and St Thomas' NHS Foundation Trust has found that the Basophil Activation Test (BAT) is more accurate than standard allergy tests for detecting cow's milk allergy in children, and could reduce the need for oral food challenges in allergy diagnosis.

177. Why we all need a 'climate safe room' (and how to create one)

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Home should be a place where people feel comfortable and safe. But the poor quality of many Australian houses means this isn't possible for some people.

178. Smartwatches allow researchers to identify human activity with 78% accuracy

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Researchers have long been able to use information from smartwatches to identify physical movement, such as sitting or walking, that wearers are performing in a controlled lab setting.

179. Sex differences in dementia: Men face a 24% higher mortality hazard

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Duke University School of Medicine-led research finds male patients with dementia have higher mortality rates and higher use of many health care services, especially hospital stays, than comparable female patients.

180. Clinical trial shows newborns with spinal muscular atrophy can start treatment at birth

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Spinal muscular atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness, which, when untreated, prevents infants with the most severe form from gaining motor development—never gaining the ability to sit—and typically leads to death before 2 years of age.

181. Scientists reverse immunotherapy-resistance by suppressing EPIC1 in mouse model of breast cancer

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Immunotherapy employs patients' own immune systems to fight cancer, and it has shown itself to be an effective treatment in many cases. However, some cancers, like triple-negative breast cancer (TNBC), show resistance to immunotherapy. This occurs when tumor cells find ways to evade immune detection—like suppressing immune signaling pathways. One such mechanism is the use of long noncoding RNAs (lncRNAs), which have been found to regulate cancer biology and immune evasion. These lncRNAs render immunotherapies, like the PD-1 inhibitor, pembrolizumab, ineffective.

182. A microbial DNA signature differentiates two types of cancer in the liver

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Determining whether a cancerous tumor originated in a given location or spread there from another tissue or organ in the body is important when assessing the likely course of a patient's disease. When the origin of the primary tumor cannot be identified, it can be challenging to properly diagnose the malignancy and determine the best treatment strategy.

183. Incorporating animals in therapy creates stronger relationships with therapists, better outcomes

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Having a furry friend in your therapy session could lead to your next big breakthrough, according to new research from Swinburne University of Technology.

184. Study finds correlation between diet and mental health

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A diet rich in vegetables and fruit is associated with reduced psychological distress, a detailed analysis of health survey data from more than 45,000 Australians has found.

185. New York City Legionnaires' disease outbreak up to 90 cases, 15 hospitalized

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There have now been 90 confirmed cases of Legionnaires' disease in central Harlem, and 15 people are currently hospitalized battling the disease, the city's health department announced.

186. FDA announces resolution of IV saline solutions shortage

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The nationwide shortage of sodium chloride 0.9% intravenous (IV) saline has officially ended, according to the U.S. Food and Drug Administration.

187. AI finds more breast tumors earlier than traditional double radiologist review

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AI is detecting tumors more often and earlier in the Dutch breast cancer screening program. Those tumors can then be treated at an earlier stage. This has been demonstrated by researchers led by Radboud university medical center in a study published in The Lancet Digital Health. The use of AI could reduce workload and save millions of euros annually.

188. Study supports evidence that prenatal acetaminophen use may be linked to increased risk of autism, ADHD

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Researchers at the Icahn School of Medicine at Mount Sinai have found that prenatal exposure to acetaminophen may increase the risk of neurodevelopmental disorders, including autism spectrum disorder and attention-deficit/hyperactivity disorder (ADHD), in children.

189. Helping others shown to slow cognitive decline

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In the latest evidence that meaningful social connections bolster health, a team from The University of Texas at Austin and University of Massachusetts Boston has found that regular time spent helping outside the home significantly slows cognitive decline in middle-aged and older adults.

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