Caciotti, Anna
Tonin, Rodolfo
Mort, Matthew
Cooper, David N.
Gasperini, Serena
Rigoldi, Miriam
Parini, Rossella
Deodato, Federica
Taurisano, Roberta
Sibilio, Michelina
Parenti, Giancarlo
Guerrini, Renzo
Morrone, Amelia https://orcid.org/0000-0003-2890-8179
Funding for this research was provided by:
BioMarin Pharmaceutical (#2052 and #2616)
Article History
Received: 11 May 2018
Accepted: 24 September 2018
First Online: 11 October 2018
Ethics approval and consent to participate
: According to local ethical guidelines, all nucleic acid samples were obtained for storage and analysis only after the patients’ family members’ written informed consent had been obtained, using a form approved by the local Ethics Committee (Ethics Committee of the Meyer Hospital, Florence, Italy).
: The patients’ family members’ written informed consents contain their permission to publish patient data.
: The authors declare that they have no competing interests.
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