The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare ...
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
LIG4 syndrome is an exceptionally rare disorder caused by a genetic mutation that prevents the body from repairing damaged ...
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and ...
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...
In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...
Also read: India’s fight against rare diseases Rare diseases, particularly chronic genetic disorders, impose life-threatening challenges, often affecting children disproportionately. Alarmingly ...
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
Carissah Holland, an Owasso girl with MPS VI, underwent multiple surgeries and advocates for others while pursuing her dreams ...
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal stimulation. Spinal stimulation restored muscles wasted by rare ...