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 ...
The fatty membrane surrounding every living cell is a vibrant environment where countless biological processes take place.
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
LIG4 syndrome is an exceptionally rare disorder caused by a genetic mutation that prevents the body from repairing damaged ...
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 ...
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 ...
Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and complex class of genetic ...
In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...
Carissah Holland, an Owasso girl with MPS VI, underwent multiple surgeries and advocates for others while pursuing her dreams ...