For a decade after its discovery, CRISPR gene editing was stuck on the cusp of transforming medicine. Then, in 2023, scientists started using it on sickle-cell disease, and Victoria Gray, a patient ...

When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...

Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to appear soon after birth. To date, there haven’t been many reliable ...

New research from the MRC Laboratory of Medical Sciences (LMS) provides a powerful, scalable method for finding treatments for rare genetic diseases using tiny, transparent worms. Subscribe to our ...

There’s a good chance that you haven’t heard about a rare condition called chromhidrosis. In a nutshell, the person afflicted with the disease produces colored sweat, from yellow to green, even red ...

Securing a diagnosis for a rare disease is a marathon, not a sprint. The search for an answer can become an odyssey of medical appointments and dead ends. But doctors diagnosed Fide Mirón’s condition ...

Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel ...