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 ...

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

A Carlsbad family is sharing their son’s story after the 5-year-old was diagnosed with a rare genetic disorder and began a gene therapy trial.

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

Blindness and deafness in some infants and sickle cell are among the conditions cured in recent gene therapy trials.

KUALA LUMPUR - A recent viral story on social media tells of a fair-skinned baby with long eyelashes who, at first glance, appears no different from any other infant. Few would have guessed that the ...

GOLDSBORO, N.C. (WITN) - A Goldsboro family is sharing their story and asking for community support as they raise their two-year-old son, diagnosed with a rare genetic disorder. Declan Newberry was ...

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

KSHB 41 reporter Olivia Acree covers portions of Johnson County, Kansas. A viewer reached out to KSHB about this story, so Olivia sat down with the family. Share your story idea with Olivia. We are ...

Add Yahoo as a preferred source to see more of our stories on Google. Max is almost six and has an extremely rare genetic disorder called TBCD - it’s often compared to ALS in babies. Late last year, ...