A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness. Doctors at ...

In a stunning medical breakthrough, scientists used customized CRISPR gene editing to save a baby’s life from a rare, previously incurable liver disorder—CPS1 deficiency—just six months after birth.

An infant in Philadelphia has been successfully treated with a customized Crispr gene-editing therapy for a rare and deadly disease, doctors reported Thursday—a breakthrough in the futuristic ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Infants with presymptomatic spinal muscular atrophy who were treated with risdiplam had improved functional and survival outcomes. At 12 months, most treated infants were able to sit and stand ...

Increasing bone density in patients with a rare genetic condition that causes bones to break easily does not prevent ...

Gaucher disease, one of the most common lysosomal storage disorders, remains widely underdiagnosed in India and globally, despite being a treatable genetic condition. Caused by a deficiency of the ...

Increasing bone density in patients with a rare genetic condition that causes bones to break easily does not prevent ...

All newborns in Manitoba will be screened for a rare genetic disorder affecting the immune system that occurs at much higher rates in this province than in the rest of the world. The provincial ...