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Treatment found to reduce progression of rare blood cancer by 74%
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Treatment found to reduce progression of rare blood cancer by 74%

Credit: Wikimedia Commons A treatment that involves genetically modifying the body’s own immune cells has been found to cut the risk of disease progression by 74 percent in people with a rare type of blood cancer, results showed Monday. Ciltacabtagene autoleucel—also known by its trade name Carvykti—was tested in a clinical trial involving 419 patients with multiple...

Scientists find treatment for rare genetic skin disorder inflammation
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Scientists find treatment for rare genetic skin disorder inflammation

by NIH/National Human Genome Research Institute Fibroblasts, shown in green, are cells that connect tissues and organs in the body and play an important role in healing wounds. Genomic variation in the STAT4 gene causes disorganized fibroblasts that fail to heal wounds properly. The nuclei of the fibroblasts are shown in blue. Credit: Ernesto Del Aguila III, NHGRI...

Pushing the boundaries of treatment for Wilson disease
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Pushing the boundaries of treatment for Wilson disease

by Serena Crawford, Yale University Uyen To, MD. Credit: Yale University Uyen To, MD, assistant professor of medicine (digestive diseases) and transplant hepatologist, discusses how she first became interested in studying Wilson disease, the wide spectrum of symptoms caused by the rare condition, and what’s next in this fascinating field of research. What is Wilson disease? Wilson...

Researchers find best treatment for excessive daytime sleepiness
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Researchers find best treatment for excessive daytime sleepiness

by McMaster University Credit: Pixabay/CC0 Public Domain McMaster University researchers Dena Zeraatkar and Tyler Pitre have found that the drug solriamfetol is the most effective treatment for excessive daytime sleepiness (EDS) for people with obstructive sleep apnea (OSA). The standard treatment for OSA is a positive airway pressure (PAP) mask that uses compressed air to support lung airways...

Bold new therapy delivery method shows initial promise as treatment for Duchenne muscular dystrophy
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Bold new therapy delivery method shows initial promise as treatment for Duchenne muscular dystrophy

CINCINNATI CHILDREN’S HOSPITAL MEDICAL CENTER IMAGE: RESEARCHERS SHOWED THAT SYSTEMIC DELIVERY OF MYMK+MYMG-LV-DYS RESULTED IN A SIGNIFICANT LEVEL OF DYS+ MYOFIBERS. IMPORTANTLY, THE TEAM DID NOT DETECT EVIDENCE OF VIRAL TRANSDUCTION IN NON-SKELETAL MUSCLE TISSUES INCLUDING HEART, KIDNEY, LIVER, AND SPLEEN. DYSTROPHIC MICE EXHIBITED HEALTHIER MUSCLES (REDUCTIONS IN MUSCLES CELLS WHERE A DYE WAS ABLE...

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Taking a placebo improves adherence to treatment for opioid use disorder, study finds

UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE Substance use disorder affects 20 million Americans, and more than 100,000 people died from a drug overdose in 2021, according to the Centers for Disease Control and Prevention. While the medication methadone has the strongest evidence-based effectiveness to prevent relapse, about half of patients drop out of their treatment within...

Prostate cancer treatment can wait for most men, study finds
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Prostate cancer treatment can wait for most men, study finds

by Carla K. Johnson  This 1974 microscope image made available by the Centers for Disease Control and Prevention shows changes in cells indicative of adenocarcinoma of the prostate. A study published by the New England Journal of Medicine on Saturday, March 11, 2023, finds long-term evidence that actively monitoring prostate cancer can be a safe...

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Rare genetic disease: Researchers discover new treatment for ADCY5-related dyskinesia

MARTIN-LUTHER-UNIVERSITÄT HALLE-WITTENBERG The movement disorder ADCY5-related dyskinesia can be treated with the asthma drug theophylline. This has been shown in a recent study by Martin Luther University Halle-Wittenberg (MLU), University Medicine Halle and University of Leipzig Medical Center. In the journal PLOS ONE, the researchers describe the case of a child with this disease whose symptoms improved...

FDA approves first treatment for geographic atrophy
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FDA approves first treatment for geographic atrophy

by Lori Solomon  Pegcetacoplan (SYFOVRE) has been approved by the U.S. Food and Drug Administration as the first and only treatment for geographic atrophy (GA), an advanced form of age-related macular degeneration. Pegcetacoplan is an injection approved for GA patients with or without subfoveal involvement and allows a flexible dosing regimen of every 25 to...

Making hepatitis C care easier when treatment seems impossible
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Making hepatitis C care easier when treatment seems impossible

by Franny White, Oregon Health & Science University Electron micrographs of hepatitis C virus purified from cell culture. Scale bar is 50 nanometers. Credit: Center for the Study of Hepatitis C, The Rockefeller University. A health care team at Oregon Health & Science University is trying a different approach to caring for patients who have,...