Miles, Lee B. http://orcid.org/0000-0002-2718-1353
Calcinotto, Vanessa http://orcid.org/0009-0002-9991-4824
Oveissi, Sara
Serrano, Rita J. http://orcid.org/0000-0002-0221-2547
Sonntag, Carmen http://orcid.org/0000-0003-2560-1710
Mulia, Orlen http://orcid.org/0009-0008-4489-6175
Lee, Clara
Bryson-Richardson, Robert J. http://orcid.org/0000-0002-9501-8208
Funding for this research was provided by:
AFM-Téléthon (23703)
Department of Health | National Health and Medical Research Council (1146321)
University of Pennsylvania Orphan Disease Center in partnership with Cure CMD
Article History
Received: 23 August 2023
Accepted: 31 May 2024
First Online: 12 June 2024
Competing interests
: The authors declare no competing interests.