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 ...

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 ...

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