ECONOMIC AND HEALTH-RELATED QUALITY OF LIFE IMPACT OF XLMTM CARERS
Research type
Research Study
Full title
A STUDY ON THE ECONOMIC AND HEALTH-RELATED QUALITY OF LIFE IMPACT OF X-LINKED MYOTUBULAR MYOPATHY (XLMTM) ON FAMILIES AND PRIMARY CARERS
IRAS ID
300888
Contact name
Asad Khan
Contact email
Sponsor organisation
Audentes Therapeutics Inc
Duration of Study in the UK
0 years, 6 months, 2 days
Research summary
The aim of this study is to further characterise the health related quality of life and economic impact of X-linked myotubular myopathy (XLMTM) on families and primary carers. Health related quality of life refers to the physical and psychological well-being of parents and primary carers while caring for participants with XLMTM. Economic impact is intended to capture as broadly as possible healthcare costs, family incurred costs, and indirect costs such as primary carer inability to work full time.
This information will be important to support the development of potential new therapies. It will also support analyses and publications that will be requested by local and global payors to enable patient access to new therapies.
REC name
London - Chelsea Research Ethics Committee
REC reference
21/PR/1020
Date of REC Opinion
4 Oct 2021
REC opinion
Further Information Favourable Opinion