clinical trials, genetics & genomics, drug development

Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

Despite a solid understanding of the biological basis of fragile X syndrome, researchers have struggled to develop effective treatments.

The agency proposes ceding its scrutiny of these studies to the FDA.

The medicine increases the activity of a deficient enzyme in certain patients with the condition.

At the annual American Association for Cancer Research meeting, researchers discuss the importance of understanding the epigenetic contributors to cancer progression and treatment response.

Sorting out which data sets are clinical-grade is key to helping patients.

A newly developed drug, modeled after a bacteria-infecting virus, is less likely to become antibiotic resistant.

A UK panel puts forth guidelines for research that use experimental animals harboring human cells and tissues.