Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships
早期使用拉罗尼酶治疗可改善轻型MPS I患者的临床结果:一项对九个同胞家庭的回顾性病例系列分析
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-015-0344-4
Al-Sannaa, Nouriya A; Bay, Luisa; Barbouth, Deborah S; Benhayoun, Youssef; Goizet, Cyril; Guelbert, Norberto; Jones, Simon A; Kyosen, Sandra Obikawa; Martins, Ana Maria; Phornphutkul, Chanika; Reig, Celia; Pleat, Rebecca; Fallet, Shari; Ivanovska Holder, Iva