Are the risks of treatment to cure a child with severe sickle cell disease too high?

Competing Interests: Competing interests: We have read BMJ policy on competing interests and declare the following: MdM has received funding for taking part in clinical trials and advisory boards from Novartis, Pfizer, and Addmedica. She has received funding to attend academic meetings from Novartis and Addmedica. VB has received funding from Addmedica for taking part in an advisory board. SC has received funding from Novartis and Pfizer for advisory boards and other meetings. AP has received funding from Bluebirdbio for attending an advisory board. JP has received funding for from Novartis, Celgene and Bluebirdbio for attending advisory boards and clinical studies. PT has received funding from Bluebirdbio, Global Blood Therapeutics and Pfizer for attending advisory boards; Novartis and Apopharma for attending meetings; and Napp and Kyora Kirin for research studies. AV has received funding from Amgen, Pfizer, Jazz Pharmaceuticals and Medac to attend academic meetings and take part in advisory boards. DR has received funding for taking part in clinical trials and advisory boards from Novartis, Eli Lilly and Bluebirdbio. He received funding to attend academic meetings from Eli Lilly, Novartis, Biogen, and Addmedica.