Rare diseases

News

Thousands of people living with rare kidney disease IgAN in the UK will soon be able to access a new oral treatment from CSL Vifor.

News

MSD's oral HIF-2 alpha inhibitor Welireg has a third FDA approval, becoming the first oral treatment for a rare solid tumour PPGL or 'pheo para'.

News

Abeona Therapeutics' Zevaskyn has been approved in the US as a treatment for the rare skin disorder recessive dystrophic epidermolysis bullosa (DEB).

News

Merck KGaA has reached an agreement with SpringWorks on a $47-per-share takeover deal that values the US biotech at $3.9 billion.

News

Pharming's Joenja has become the first disease-modifying drug that can be used by the NHS to treat APDS, an ultra-rare immunodeficiency disorder.

News

Spruce has bounced back from the failure of its lead drug last year, thanks to a licensing deal with BioMarin for a late-stage Sanfilippo drug.