[1] Yale Sch Med, Dept Internal Med & Pediat, New Haven, CT USA
[2] Icahn Sch Med Mt Sinai, Dept Genet & Genom Sci, New York, NY 10029 USA
[3] Univ Miami, Miller Sch Med, Dept Human Genet, Coral Gables, FL 33124 USA
[4] Univ Arkansas Med Sci, Dept Pediat, Little Rock, AR 72205 USA
[5] Arkansas Childrens Hosp, Sect Genet & Metab, 800 Marshall St, Little Rock, AR 72202 USA
[6] Univ Massachusetts, Sch Med, Dept Psychiat, Lysosomal Disorders Treatment & Res Program, Worcester, MA 01655 USA
[7] Univ Massachusetts, Sch Med, Dept Neurol, Lysosomal Disorders Treatment & Res Program, Worcester, MA USA
[8] Lysosomal & Rare Disorders Res & Treatment Ctr, Fairfax, VA USA
[9] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp Med Ctr, Dept Pediat, Cincinnati, OH USA
[10] Univ Cincinnati, Coll Med, Cincinnati Childrens Hosp Med Ctr, Dept Mol Genet & Biochem, Cincinnati, OH USA
[11] Univ Minnesota, Dept Expt & Clin Pharmacol, Ctr Orphan Drug Res, Minneapolis, MN USA
[12] Duke Univ, Med Ctr, Dept Pediat, Div Med Genet, Durham, NC 27710 USA
[13] NYU, Dept Neurol, Div Neurogenet, New York, NY 10016 USA
[14] Stanford Univ, Lucile Packard Childrens Hosp Stanford, Div Med Genet, Dept Pediat,Sch Med, Palo Alto, CA USA
[15] NHGRI, Med Genet Branch, NIH, Bldg 35A Room 1E623,35A Convent Dr,MSC 3708, Bethesda, MD 20892 USA
[16] Univ Florida, Dept Pediat, Div Genet & Metab, Gainesville, FL USA
[17] Univ Florida, Dept Neurosci, Div Genet & Metab, Gainesville, FL 32610 USA
[18] Univ Florida, Dept Mol Genet & Microbiol, Div Genet & Metab, Gainesville, FL USA
[19] Univ Calif San Francisco, Dept Pediat, Div Med Genet, San Francisco, CA USA
[20] Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati Childrens Hosp Med Ctr,Div Human Genet, Cincinnati, OH USA
[21] Cedars Sinai Tower Hematol Oncol, Beverly Hills, CA USA
[22] Childrens Natl Med Ctr, Rare Dis Inst, Washington, DC 20010 USA
[23] Baylor Scott & White Res Inst, Dallas, TX USA
[24] Univ Miami, Miller Sch Med, Dept Human Genet, Miami, FL 33136 USA
[25] Univ Miami, Miller Sch Med, Dept Internal Med, Miami, FL 33136 USA