Why rare diseases are an important medical and social issue

被引：403

作者：

Schieppati, Arrigo ^{[1
]}

Henter, Jan-Inge ^{[2
]}

Daina, Erica ^{[1
]}

Aperia, Anita ^{[2
]}

机构：

[1] Ist Ric Farmacol Mario Negri, Clin Res Ctr Rare Dis Aldo & Cele Dacco, I-24020 Bergamo, Italy

[2] Astrid Lindgren Childrens Hosp, Dept Women & Child Hlth, Karolinska Inst, Stockholm, Sweden

来源：

关键词：

D O I：

10.1016/S0140-6736(08)60872-7

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

[No abstract available]

引用

页码：2039 / 2041

页数：3

共 12 条

[1] Inborn errors of metabolism in the Italian pediatric population: A national retrospective survey
Dionisi-Vici, C
Rizzo, C
Burlina, AB
Caruso, U
Sabetta, G
Uziel, G
Abeni, D
[J]. JOURNAL OF PEDIATRICS, 2002, 140 (03) : 321 - 327
[2] Eurordis, EURORDISCARE2 SURV D
[3] Focus on research - Adopting orphan drugs - Two dozen years of treating rare diseases
Haffner, ME
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 2006, 354 (05) : 445 - 447
[4] *IND EXP GROUP, 2004, ETH LEG SOC ASP GEN
[5] *ORPH, RAR DIS NUMB PREL RE
[6] *ORPHANXCHANGE, ORPH DRUGS
[7] *US DEP HHS, 1989, REP NAT COMM ORPH DI
[8] VANWEELY S, PRIORITY MED EUROPE
[9] A journey of hope:: lessons learned from studies on rare diseases and orphan drugs
Wästfelt, M.
Fadeel, B.
Henter, J. -I.
[J]. JOURNAL OF INTERNAL MEDICINE, 2006, 260 (01) : 1 - 10
[10] WEINER DL, PEDIAT INBORN ERRORS