[1] Nemours Childrens Hosp, Orlando, FL USA
[2] St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA
[3] Columbia Univ, Irving Med Ctr, New York, NY USA
[4] Univ Toronto, Hosp Sick Children, Toronto, ON, Canada
[5] Stanford Univ, Sch Med, Stanford, CA USA
[6] Ionis Pharmaceuticals, Carlsbad, CA USA
[7] Locana, San Diego, CA USA
[8] Biogen, Maidenhead, Berks, England
[9] Biogen, Cambridge, MA USA