News NICE relents and backs drug for IgAN from CSL Vifor 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 adds new indication for Welireg in rare tumours 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 prices rare cell disorder gene therapy at $3.1m Abeona Therapeutics' Zevaskyn has been approved in the US as a treatment for the rare skin disorder recessive dystrophic epidermolysis bullosa (DEB).
News Merck agrees $3.9 billion takeover of SpringWorks 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 First drug for rare disease APDS cleared for NHS use 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 Bio sows the seeds of its new future 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.
News RFK Jr spreading 'harmful myths' on Alzheimer's research The Alzheimer's Association has hit out at "inaccurate and dangerous fabrications" by HHS Secretary Robert F Kennedy Jr about NIH dementia research.
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