Patients suffering from x-linked myotubular myopathy (XLMTM) do not have enough of the myotubularin protein, so the body may require a ventilator to support breathing and a feeding tube.

Gene Therapy, the first treatment candidate for XLMTM, is explained here on the ASGCT website and in an educational video.

What exactly is gene therapy? How might it work? A new, easy-to-understand education resource – Gene Therapy 101 – has just been published by the American Society of Gene & Cell Therapy to explain the basics of this timely topic.  With the help of a number of patient groups, including the Myotubular Trust, ASGCT have created some great visuals and clear wording, to outline what it means to try to change a gene, why viruses are used, the steps required to develop a treatment for a rare disease. We welcome this clear explanation of a topic that is of great interest to so many of us.Please visit the Gene Therapy 101 website here.

Download the leaflet here: ASGCT Patient Education Leaflet (pdf)

More about the ASGCT’s Patient Education programme: This is new initiative for 2019. The new patient-centered portal is designed by ASGCT committee volunteers in coordination with patient advocacy groups to educate and inform patients, families, and the general public on the status and promise of gene and cell therapies.