Thompson, Rachel
Straub, Volker
Article History
First Online: 1 April 2016
Competing interests
: V.S. is or has been a principal investigator for trials sponsored by Genzyme, GSK, Prosensa/Biomarin, ISIS Pharmaceuticals, and Sarepta. He has received speaker honoraria from Genzyme and has been a member of the company's international Pompe advisory board. He also is or has been on advisory boards for Acceleron Pharma, Audentes Therapeutics, Bristol-Myers Squibb, Italfarmaco, Nicox, Pfizer, Prosensa, Santhera, Summit Therapeutics and TrophyNOD. He has research collaborations with Ultragenyx pharmaceutical and Genzyme/Sanofi. None of the industry collaborations pose a conflict of interest for this Review article. R.T. declares no competing interests.