Calling families and primary carers of children with XLMTM to take part in a new study about the ‘financial impact and quality of life of XLMTM’

Have you ever been the primary carer for someone with X-linked myotubular myopathy (XLMTM)?

If you have, we wish to invite you to take part in a new study.

Most patients with XLMTM have severe physical limitations that require substantial support from families and carers, but this burden is not fully understood.

Astellas Gene Therapies is conducting the ‘Families and Primary Carers Economic and Quality of Life Impact of XLMTM Survey‘ to help improve understanding and raise awareness of the physical, social, emotional, and financial impact of XLMTM on families and primary carers in the UK.

The survey will be open for 6 months and up to 20 primary carers of those with XLMTM may participate.

Results from this survey will be: shared with the XLMTM community, including the Myotubular Trust, as well as health policy makers, and NICE; presented at an upcoming neuromuscular conference; and submitted for publication in a scientific journal in Spring of 2024. This information may also be used to help to support patient access to potential treatments. For additional questions, please e-mail us at AGT_medinfo@astellas.com.

The survey webpage will provide further information. To get started, go to the following link: https://qrco.de/bd2DDe 

Download the XLMTM Families and Primary Carers Survey (UK) Brochure (PDF).

Thank you!