Ten-year-old Sebastian Veiman is racing against time to receive €2.5 million gene therapy that could halt — or even reverse — ...

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” study, according to drugmaker uniQure, marking a major step toward a potential first genetic ...

Since we last saw Lo, she has hit a lot of milestones. Lo, 5, just started kindergarten and her parents said her ...

Winsights.life envisions a HIPAA-protected space where families own their health data, track their child's progress, and ...

While sentiment in the local biotech sector is improving, a plethora of smaller revenue-generating stocks remain deeply ...

A Columbus family is on a mission to find a cure for a rare genetic disorder. Saturday morning was their third annual Hope With Lo 5K. “We believe that hope isn’t a feeling that it’s a force. We’ve ...

The seventh annual Calves to Cure DMD charity cattle auction raised funds to help fund research for Duchenne muscular ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE ...

Mohiuddin said voices of the autistic community should come first when establishing priorities for research and treatment.

It turned out Casteneda had a rare vaginal cancer called clear cell carcinoma. Thanks to her insistence, she was diagnosed a month before her 15th birthday. In this condition, the cancer cells look ...

A Harvard Medical School–led research team has developed an AI tool that can reliably tell apart two look-alike cancers found ...