New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options.

Conducted as part of the multicenter RUBY Trial, researchers published their latest findings in the New England Journal of Medicine. Remarkably, 27 out of 28 patients did not have any painful sickle cell crises after treatment, achieving what physicians call a "functional cure."

Being unable to afford dental care may increase a person’s risk of developing cardiovascular disease or dementia, according to a new study led by researchers at Boston University School of Public Health. Published in The Journals of Gerontology, Series A, the study found that older people who forego necessary dental procedures because of financial barriers may be more likely to experience heart failure, a heart attack, a stroke, or dementia.

Understanding gun violence risk factors, along with effective policies and interventions, can help reverse the alarming rate of global gun violence—but researchers face multiple challenges in achieving these goals, according to a new commentary published in The Lancet. 

The small molecule could slow or stop the progression of Parkinson’s disease and related brain disorders, rather than just treating symptoms.

The drug candidate works by blocking and reversing the toxic build-up of protein believed to drive these diseases, preventing it from spreading through the brain.

The compound is one of the first to target multiple disease pathways at once, including the interaction between key proteins linked to faster cognitive decline.

In some non-small cell lung cancers (NSCLCs), changes to the RET gene (known as RET fusions) can drive tumor growth. In a phase 1/2 clinical study with a 42-month-long follow-up period, researchers from Mass General Brigham Cancer Institute evaluated the long-term efficacy and safety of the FDA-approved drug pralsetinib, which targets RET. Investigators found that treatment led to durable responses with manageable safety profiles in 281 patients with advanced or metastatic RET fusion-positive NSCLCs. Results are published in Journal of Clinical Oncology.