Saou Ichikawa’s award-winning novel, “Hunchback,” is narrated by an heiress with a rare genetic disorder and a brilliant, ...

The write-down comes in the wake of problems affecting the Japanese drugmaker's AT132 gene therapy candidate for rare disease X-linked myotubular myopathy (XLMTM), which was placed on clinical ...

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 safety ...

Elijah Cariazo, 13, was born with a rare muscular condition called myotubular myopathy. He has difficulty breathing and swallowing and needs the grey Ford Transit Custom to get to hospital ...