New Anti-Blood Clotting Drug May Lower Risk of Recurrent Strokes

An experimental drug designed to block blood-clotting proteins may lower the risk of recurrent strokes, according to a dose-finding trial published in The Lancet Neurology. More than 795,000 people in the United States each year suffer a stroke, according to the American Heart Association, and nearly one in five will go on to experience another stroke. “When patients

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Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant, the first to receive gene therapy in the United States for a genetic form of hearing loss AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF)

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New research finds half-cardio, half-strength training reduces cardiovascular disease risks

Approximately one in three deaths in the U.S. is caused by cardiovascular disease, according to the U.S. Centers for Disease Control and Prevention. A robust body of evidence shows aerobic exercise can reduce risks, especially for people who are overweight or obese. But few studies have compared results with resistance exercise — also known as strength

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The cause of Guillain-Barré syndrome elucidated

Guillain-Barré syndrome is a rare disease in which the immune system attacks the peripheral nerves. Those affected suffer from muscle weakness and paralysis. A research team led by ETH Zurich has now elucidated the mechanism of this autoimmune reaction. Patients with Guillain-Barré syndrome (GBS) suffer from a rare and diverse disorder of the peripheral nervous

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First-in-human clinical trial of CAR T cell therapy with new binding mechanism shows promising early responses

Improving CART19 function by targeting a membrane-proximal CD19 epitope with fast on- and off-rates. Early results for the Penn Medicine-developed AT101 presented at ASH Early results from a Phase I clinical trial of AT101, a new CAR T cell therapy that uses a distinct binding mechanism to target CD19, show a 100 percent complete response

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FDA Approves First CRISPR-based Gene Therapies to Treat Patients with Sickle Cell Disease

The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an

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Personalized cancer medicine: humans make better treatment decisions than AI

Charité study highlights limits of large language models in precision medicine Treating cancer is becoming increasingly complex, but also offers more and more possibilities. After all, the better a tumor’s biology and genetic features are understood, the more treatment approaches there are. To be able to offer patients personalized therapies tailored to their disease, laborious

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