An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

A man is set to take on one of the world’s toughest triathlons to raise money for a friend’s son. Sam Weale, 44, from Monmouth, will attempt the Enduroman Arch 2 Arc triathlon this August in support ...

A new investigation into Duchenne muscular dystrophy (DMD) pathogenesis suggests that at least part of the muscle degeneration observed in DMD patients may result from the reduced production of ...

Targeted rewriting of the epigenome promises to overcome several challenges in direct gene editing in patient therapies.

What has Wall Street been buzzing about this week? Here are the top 5 Buy calls and the top 5 Sell calls made by Wall Street’s best analysts ...

In a striking bipartisan move, the Senate Appropriations Committee voted 26-3 to boost the NIH’s budget by $400 million — rejecting the Trump administration’s proposed $17 billion cut and sweeping ...

Prasad, a longtime critic of the FDA’s leniency in drug approvals, joined the agency in May. WASHINGTON — The Food and Drug ...

Dr George Tidmarsh, a former pharmaceutical executive who joined the FDA this month, will take over Prasad’s role temporarily.

Columnist Betty Vertin, whose days often feel overloaded, describes how it feels when her life as a caregiver is recognized and affirmed.