Hari Jones from Caernarfon in Gwynedd has myotubular myopathy, which means the muscles he uses to breathe and swallow do not work. Merseyside Police gave him a guard of honour as he left on Tuesday.
In a study of gene therapy for X-linked myotubular myopathy, patients required high doses of the adeno-associated viral (AAV) vector to ensure that enough genetic material could reach the skeletal ...
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 ...
This is one of thousands of bunting flags that were made into the world's longest line (now in the world records) to raise money for myotubular myopathy, which my best friend's brother has.