Agrawal, Nidhi
Mehta, Sonal
Feelders, Richard A.
Skwiersky, Samara
Campana, Claudia
Dogan, Fadime
van Koetsveld, Peter M.
Neggers, Sebastian J. C. M. M.
Wright, Kyla
Kim, Hyon
Zagzag, David
Hofland, Leo J.
Funding for this research was provided by:
Recordati Rare Diseases
Article History
Received: 16 July 2025
Accepted: 5 September 2025
First Online: 3 November 2025
Declarations
:
: The authors declare no competing interests.