Funding for this research was provided by:
BioMarin Pharmaceutical (#2052 and #2616)
Text and Data Mining valid from 2018-10-11
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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