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Article History

Received: 3 July 2018

Accepted: 21 September 2018

First Online: 4 October 2018

Authors’ information

: AA, Vice-President of the ADR; member of the NMD Alliance Board.DC, CMT and SBMA Registries’ Curator who prepared the Clinical SOPs of the Registries.FMA, Chair of the ELAC of the ADR.FC, IT consultant and developer.GC, Chair of the NMD Alliance Board.MCP, Curator of the DMD and SMA registries.AT, Scientific Coordinator of the MGSD registry and representative of the GMSD Scientific Committee.GV, Scientific Coordinator of the TTR-FAP Registry and representative of the TTR-FAP Scientific Committee.LM, RD-Connect Biobanking Workpackage Coordinator; member of the NMD Alliance Board.DP, Scientific Coordinator of the CMT and SBMA registries and representative of the CMT and SBMA Scientific Committees; member of the NMD Alliance Board

: All individuals who participate in any of the registries reported in the text did register (or approved registration by their referring clinician) on voluntary base and signed an ad hoc Informed Consent. The protocol of the DMD/BMD and SMA registries and the Italian version of the Informed Consent have been approved on July 28, 2009 by the <i>ADR – ELAC,</i> composed by Francesco Maria Avato (Ferrara, author); Sara Casati (Milan); Alessandro Martini (Padua); Deborah Mascalzoni (Bolzano); Livio Tronconi (Pavia).All documents related to clinician-driven registries received approval from the Coordination Centres’ IRBs; subsequently, the IRBs of all clinical centres involved in each registry were notified about the approval and their approval followed (All names of the Institutes of the IRBs are reported in the Additional file , which lists the affiliation of all Working groups’ participants). For the sake of simplicity, only the information on the Coordination centres’ IRB and date/No. of approval is reported below:

: Not applicable.

: DP is member of the Clinical Advisory Board of Inflectis BioSciences; AT has received honoraria from Sanofi Genzyme and CSL Behring for educational purposes and participation as a component of the Pompe Registry Board; GV is member of the Clinical Advisory Board of Pfizer Italia, Alnylam Pharmaceutics, and Avexis Pharm. All other authors declare that they have no competing interests.

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