Rare Disease

News

Astellas reports fourth death in halted gene therapy trial

There has been a fourth patient fatality in Astellas' clinical trial of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), which has been halted twice due to

News

DNA helix in a futuristic concept of the evolution of science and medicine.

FDA slaps clinical hold on BioMarin's PKU gene therapy

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

News

More trouble for Astellas as AT132 gene therapy trial is hal...

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

News

After Ultomiris stumble, Alexion delivers a rare disease win...

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.

News

NICE says yes to Novartis' Rydapt for rare blood disorder

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

News