Rare diseases

News

AstraZeneca's light chain (AL) amyloidosis candidate anselamimab has failed a phase 3 readout, but showed efficacy in a patient subgroup.

News

LEO Pharma has licensed a medicine for a rare form of psoriasis from Boehringer Ingelheim, plugging a gap in its near-term product portfolio.

News

KalVista has brought the first oral, on-demand treatment for hereditary angioedema attacks to the US market.

News

LifeArc and Genetics Alliance UK have issued a call to the government to remove obstacles impeding R&D into rare diseases.

R&D

Dr Bruce Leuchter, CEO of GRIN Therapeutics and Neurvati Neurosciences, discusses the companies GRIN-NDD work with pharmaphorum.

News

Neopharmed Gentili buys BioCryst's hereditary angioedema drug Orladeyo in Europe, the first stage of an international expansion for the Italian firm.