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

The early stage company is developing a new approach to gene editing to treat rare genetic diseases. It does this using mRNA, ...

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

A new study shows that long-read sequencing has the potential to improve the rate of diagnosis while reducing the time to diagnosis from years to days -- in a single test and at a much lower cost.

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

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

AI-driven genomics startups leverage technology to analyze genetic data, predict predispositions, and offer personalized ...

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 ...

Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...

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

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