Two Johns Hopkins Medicine faculty members have been elected to the National Academy of Medicine (NAM), an independent ...

Biotech stocks rebound after years of weakness as M&A, lower rates and easing policy fears draw investors back to the sector.

In an interview-based study, adults with SMA reported a lack of coordination during their transition from pediatric care once ...

The crucial funds will help to cover some of the costs required to run Noah’s Ark Children’s Hospice, which currently stands ...

The Muscular Dystrophy Association (MDA) today kicked off its largest annual MDA Holiday Retail Campaign, spanning thousands of retail ...

Expatriates in Kuwait often face complex rules when it comes to bringing their children under family (dependent) visas. With ...

Biogen specializes in therapies for complex neurological and neurodegenerative conditions. With a diversified product suite ...

In a case that was billed as profits versus patients, India’s Supreme Court dismissed a petition in which Roche sought to ...

The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...

Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...

Today, Sammy is 17 years old and working hard to be able to walk across the stage at her high school graduation next spring. Whether kids like Sammy can achieve their dreams depends on whether ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...