In unusual circumstances, Ipsen has been given the green light for its odevixibat drug for severe itching (pruritus) in patients with rare liver disease Alagille syndrome (ALGS) in the EU –
Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the
An experimental gene therapy developed by Lexeo Therapeutics has shown early promise in treating one of the most serious complications of the rare neurodegenerative disorder, Friedreich's a
KalVista Pharma has filed its first marketing application to the US FDA, seeking approval for its oral plasma kallikrein inhibitor sebetralstat as an oral treatment for hereditary angioedem
Amgen has moved closer to a second indication for its B cell-depleting therapy Uplizna, acquired as part of its $27.8 billion takeover of Horizon Therapeutics last year, a