"This type of gene editing should also allow for the treatment of diseases whose origin is not genetic or whose origin cannot ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...

Study highlights use of techniques such as long-read genome sequencing, optical genome mapping, and RNA sequencing for rare ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

A teenager diagnosed with a rare genetic eye disease which means she will lose her eyesight is desperate to see the world ...

After more than a year the HSHS Hospital/Prevea Clinic closures were announced, a couple of Eau Claire County boys have ...

One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid increases in genetic technology and testing.

The healthcare industry is set for growth due to advancements in targeted treatments and personalized care and the rapid ...

Ultragenyx Pharmaceutical is a mid-cap biotech firm with a promising pipeline and an optimistic outlook from analysts. Click ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

This case study explores a rare overlap of hidradenitis suppurativa and porokeratosis, as well as highlights the unique ...