The treatment landscape of Inflammatory Bowel Disease (IBD) has undergone a significant transformation over the past two ...

Around 75% of rare diseases are diagnosed in childhood, with most before the age of two. With 30% of rare disease patients ...

NICE's initial guidance on Breyanzi concluded it was not a cost-effective option for patients, but the agency said this ...

Pharma industry leaders are scheduled to meet with Donald Trump later today to discuss a range of issues affecting the sector ...

A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...

AstraZeneca and Daiichi Sankyo's HER2-directed antibody-drug conjugate Enhertu (trastuzumab deruxtecan) already has ...

The drug is the first non-cannabinoid option for patients with LGS and an alternative to Jazz Pharma's Epidyolex (cannabidiol ...

Biogen has bolstered its pipeline by licensing ex-US rights to a drug for Dravet syndrome – a rare form of epilepsy – from ...

Bullous pemphigoid (BP), which has no FDA-approved targeted therapies, leads to reddening of the skin and the formation of ...

Four more experimental drugs for amyotrophic lateral sclerosis (ALS) have failed to hit the primary endpoint in the phase 2/3 ...

Analysts at RBC Capital Markets have predicted twice-yearly lenacapavir could become a $2 billion product, and become a key ...

A lawsuit filed in the Singapore International Commercial Court is seeking $830 million in damages from KBP, with Novo ...