Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

[1] Schey C(2011)Estimating the budget impact of orphan medicines in Europe: 2010-2020 Orphanet J Rare Dis 6 62-e281

[2] Milanova T(2009)Evidence and values: requirements for public reimbursement of drugs for rare diseases–a case study in oncology Can J Clin Pharmacol 16 e273-432

[3] Hutchings A(2007)Coverage with evidence development: an examination of conceptual and policy issues Int J Technol Assess Health Care 23 425-30

[4] Drummond M(2009)Access mechanisms for orphan drugs: a comparative study of selected European countries Off Health Econ 52 1-S43

[5] Evans B(2004)Pompe disease in infants and children J Pediatr 144 S35-1353

[6] LeLorier J(2008)Pompe’s disease Lancet 372 1342-716

[7] Karakiewicz P(1999)Frequency of glycogen storage disease type II in the Netherlands: implications for diagnosis and genetic counselling Eur J Hum Genet 7 713-340

[8] Martin D(2003)The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature Pediatrics 112 332-676

[9] Tugwell P(2006)A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease J Pediatr 148 671-109

[10] MacLeod S(2007)Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease Neurology 68 99-335

[1] Schey C(2011)Estimating the budget impact of orphan medicines in Europe: 2010-2020 Orphanet J Rare Dis 6 62-e281

[2] Milanova T(2009)Evidence and values: requirements for public reimbursement of drugs for rare diseases–a case study in oncology Can J Clin Pharmacol 16 e273-432

[3] Hutchings A(2007)Coverage with evidence development: an examination of conceptual and policy issues Int J Technol Assess Health Care 23 425-30

[4] Drummond M(2009)Access mechanisms for orphan drugs: a comparative study of selected European countries Off Health Econ 52 1-S43

[5] Evans B(2004)Pompe disease in infants and children J Pediatr 144 S35-1353

[6] LeLorier J(2008)Pompe’s disease Lancet 372 1342-716

[7] Karakiewicz P(1999)Frequency of glycogen storage disease type II in the Netherlands: implications for diagnosis and genetic counselling Eur J Hum Genet 7 713-340

[8] Martin D(2003)The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature Pediatrics 112 332-676

[9] Tugwell P(2006)A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease J Pediatr 148 671-109

[10] MacLeod S(2007)Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease Neurology 68 99-335