Chen, Brenden
Whatley, Sharon
Badminton, Michael
Aarsand, Aasne K.
Anderson, Karl E.
Bissell, D. Montgomery
Bonkovsky, Herbert L.
Cappellini, Maria D.
Floderus, Ylva
Friesema, Edith C.H.
Gouya, Laurent
Harper, Pauline
Kauppinen, Raili
Loskove, Yonina
Martásek, Pavel
Phillips, John D.
Puy, Hervé
Sandberg, Sverre
Schmitt, Caroline
To-Figueras, Jordi
Weiss, Yedidyah
Yasuda, Makiko
Deybach, Jean-Charles
Desnick, Robert J.
Funding for this research was provided by:
Icahn School of Medicine at Mount Sinai
Article History
Received: 28 February 2019
Accepted: 26 April 2019
First Online: 10 May 2019
Disclosure
: K.E.A., R.J.D., and J.D.P. are consultants for Alnylam Pharmaceuticals, Recordati Rare Diseases, Mitsubishi Tanabe Pharma. R.J.D. and M.Y. are inventors of intellectual property licensed to Alnylam Pharmaceuticals. K.E.A., R.J.D., and M.Y. have received research grants from Alnylam Pharmaceuticals and Recordati Rare Diseases. J.-C.D. is a consultant for Alnylam Pharmaceuticals and Orphan Europe Recordati Group. H.L.B. is a consultant for Alnylam Pharmaceuticals, Recordati Rare Diseases, and Moderna Therapeutics, Inc. and has received research grants from Alnylam Pharmaceuticals and Gilead Sciences, Inc. E.C.H.F. has received lodging support from Alnylam Pharmaceuticals. The respective honorarium from Alnylam for participating in an advisory group has been paid to Erasmus MC. A.A.K. has received travel and accommodation support from Clinuvel Pharmaceuticals. L.G. has received travel and lodging support from Alnylam Pharmaceuticals and Clinuvel Pharmaceuticals (no honorarium). P.H. has received travel and lodging support from Alnylam Pharmaceuticals and Clinuvel Pharmaceuticals. The respective honorarium from Alnylam for participating in an advisory group has been paid to Karolinska University Hospital or Karolinska Institutet. R.K. received travel and lodging support from Alnylam Pharmaceuticals and Clinuvel Pharmaceuticals and holds stock in Orion Pharma. S.S. received an honorarium from Alnylam for participating in an advisory group. J.T.-F. was compensated for limited in time consulting services by Moderna Therapeutics and Alnylam Pharmaceuticals Spain, SL. This work was primarily supported by the Department of Genetics and Genomic Sciences of the Icahn School of Medicine at Mount Sinai. K.E.A., D.M.B., H.L.B., R.J.D., and J.D.P. receive salary support from the Porphyrias Consortium (U54DK083909), which is part of the National Center for Advancing Translational Sciences (NCATS) Rare Diseases Clinical Research Network (RDCRN) of the National Institutes of Health (NIH). RDCRN is an initiative of the Office of Rare Diseases Research (ORDR), NCATS, funded through collaboration between NCATS and the National Institute for Diabetes and Digestive and Kidney Diseases (NIDDK). The other authors declare no conflicts of interest.