Acknowledgements
RARE DISEASE
This study was funded by Alexion, AstraZeneca Rare Disease. The authors thank Sivani Paskaradevan and Manoj Rajadhyaksha (Alexion, AstraZeneca Rare Disease) for critical review of the manuscript. Editorial support was provided by Duncan Porter of Piper Medical Communications, funded by Alexion, AstraZeneca Rare Disease.