Abstract

AADC-d is an ultra-rare autosomal recessive neurometabolic disorder leading to hypotonia, movement disorders, developmental delay and autonomic symptoms from birth. Given the lack of healthcare resource utilization (HCRU) information, the aim of this study, part of a global burden of disease project, was to generate French data to feed a cost-effectiveness model to support HTA submission.

Talk to us

Join us for a 30 min session where you can share your feedback and ask us any queries you have

Schedule a call

Disclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.