Archives: 2024-03-19

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).  Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. It is caused by a

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Mayo Clinic researchers find promise in new potential treatment for liver failure

A new drug has been shown to increase healing and regeneration of the liver after major surgery, according to a study published in the scientific journal Cell. Researchers hope that this could lead to more surgical options for patients diagnosed with advanced liver tumors and liver failure. “This research is significant because this is the first

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A micro-fragmented collagen gel as a stem cell-assembling platform for critical limb ischemia repair

Critical limb ischemia is a condition in which the main blood vessels supplying blood to the legs are blocked, causing blood flow to gradually decrease as atherosclerosis progresses in the peripheral arteries. It is a severe form of peripheral artery disease that causes progressive closure of arteries in the lower extremity, leading to the necrosis

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Preliminary Clinical Trial Results Show ‘Dramatic and Rapid’ Regression of Glioblastoma after Next Generation CAR-T Therapy

A collaborative project to bring the promise of cell therapy to patients with a deadly form of brain cancer has shown dramatic results among the first patients to receive the novel treatment. In a paper published today in The New England Journal of Medicine, researchers from the Mass General Cancer Center, a member of the Mass

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Fibrinolytic Biomarkers for Identifying Patients at Risk of Severe COVID-19

Researchers identify associations between proteins involved in fibrinolysis and the development of acute respiratory distress syndrome During the peak of the COVID-19 pandemic, healthcare services experienced an overburdening surge in admissions. Recognizing the complexity of managing COVID-19 cases, researchers have identified proteins related to fibrinolysis as potential biomarkers for evaluating the risk of a patient

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Zilebesiran RNAi therapeutic lowers blood pressure for months

The results of a phase II study with the RNAi therapeutic zilebesiran have appeared in the specialist journal “JAMA”. Accordingly, injections of the drug every three or every six months lower blood pressure sustainably. Zilebesiran is a substance that exerts its pharmacological effect via RNA interference (RNAi) . The active ingredient is a short RNA section that, as small

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Simple Blood Protein Tests Predict Which Lymphoma Patients Are Most Likely to Have Poor CAR T Outcomes

International research team develops, validates approach for assessing and responding to elevated risk As new cancer treatments become available, some of the most important ongoing research must look at ways to optimize those new approaches so that more patients can benefit from groundbreaking therapies. In work newly published in Blood Cancer Discovery, a journal of the American Association

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