Stein, Susan
Bogard, Elizabeth
Boice, Nicole
Fernandez, Vivian
Field, Tessa
Gilstrap, Alan
Kahn, Susan R.
Larkindale, Jane
Mathieson, Toni
Article History
Received: 12 October 2017
Accepted: 4 January 2018
First Online: 22 January 2018
Ethics approval and consent to participate
: Not applicable.
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: SS is President and Chief Executive Officer of Connexion Healthcare, a medical communications company and a Board Member of Global Genes.EB is Chair of the Research Committee of the International Fibrodysplasia Ossificans Progressiva Association (IFOPA).NB is Founder and Chief Executive Officer of Global Genes.VF is Director of Patient Advocacy at REGENXBIO Inc.TF is Patient Advocacy Lead at Spark Therapeutics.AG is Executive Director of Advocacy and Policy at Akcea Therapeutics.SRK is Executive Director of the National Tay-Sachs & Allied Diseases Associations Inc.JL is Vice President of Research Development at the Friedreich’s Ataxia Research Alliance and Executive Director of the Duchenne Regulatory Science Consortium at the Critical Path Institute.TM is Chief Executive, Niemann-Pick UK (NPUK); UK Representative, International Niemann-Pick Disease Alliance (INPDA); NPUK Representative, UK LSD Collaborative; and Patient/Public Voice (PPV) Member, NHS England Clinical Reference Group for Inherited Metabolic Disease; and Member of the UK LSD Expert Advisory Group.
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