Lo, Ph.D., Professor at the MIT Sloan School of Management and a member of n-Lorem’s board of directors, have published a commentary in Nature Biotechnology that proposes more economically feasible ...

Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

Natera NTRA announced the commercial launch of Zenith genomics, a next-generation whole genome sequencing assay designed to ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

Living with a rare disease often means years of uncertainty, misdiagnosis, and limited treatment options. But advances in ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. The baby, KJ Muldoon of Clifton Heights, ...

Jack Arneson, born Jan. 26, has both Ebstein's anomaly, a congenital heart defect, and Sotos syndrome, a rare genetic disorder.

An Ohio man born with a rare genetic disorder that leaves skin as fragile as a butterfly’s wing has helped inspire federal ...

Non-invasive prenatal testing (NIPT) is a simple blood test during pregnancy that helps detect rare genetic conditions early, ...

Sildenafil—an active ingredient also marketed under the name of Viagra—improves symptoms in patients with Leigh syndrome.

Connecticut prides itself on strong schools, world-class healthcare, and a commitment to caring for its most vulnerable residents. We are a state that values education, innovation, and community. Yet ...

Maria Baum was born with Williams syndrome, a genetic neurodevelopmental disorder strongly linked to cardiovascular disease ...