BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PK
Astellas has halted dosing in a study of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM) for the second time after another serious adverse event (SAE) linke
A drug for Wilson disease developed by AstraZeneca's Alexion rare disease unit has cleared a phase 3 trial, setting up regulatory filings in the coming months.
Novartis' Rydapt has become the first and only licensed treatment for rare and life-threatening blood disorder systemic mastocytosis (SM) to be cleared for routine NHS use, after getting a
Merck & Co has extended its range of cancer medicines in the US after claiming FDA approval for Welireg – a drug it acquired as part of its $2.2 billion takeover of Peloton Therapeutics