European regulators have granted a fast path to market for Calliditas Therapeutics’ potential rare kidney disease drug, Nefecon, weeks after a similar decision by the FDA.
Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene therapy Zolgensma to a seriously ill child in Belgium, saying compassionate use rules do
bluebird bio has produced updated data from its gene therapy for the rare genetic neurological disease cerebral adrenoleukodystrophy (CALD), showing that it continues to work in the long-te
Patients in England and Wales with hereditary angioedema (HAE) should soon have a new treatment option after NICE recommended Takeda’s Takhzyro (lanadelumab) should be funded by the NHS in