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

Van der Ploeg AT(2008)Pompe's disease Lancet 372 1342-1353

Reuser AJJ(2005)The natural course of non–classic Pompe’s disease; a review of 225 published cases J Neurol 252 875-884

Winkel LPF(2005)Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients Brain 128 671-677

Hagemans MLC(1999)Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling Eur J Hum Genet 7 713-716

Van Doorn PA(2011)Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy Orphanet J Rare Dis 6 34-1052

Loonen MCB(2011)Burden of illness of Pompe disease in patients only receiving supportive care J Inherit Metab Dis 34 1045-1692

Hop WJC(2004)Late-onset Pompe disease primarily affects quality of life in physical health domains Neurology 63 1688-1406

Reuser AJJ(2017)European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience Eur J Neurol 24 768-260

Hagemans MLC(2010)A randomized study of alglucosidase alfa in late-onset Pompe's disease N Engl J Med 362 1396-398

Winkel LPF(2016)Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up J Inherit Metab Dis 39 253-109

Van der Ploeg AT(2008)Pompe's disease Lancet 372 1342-1353

Reuser AJJ(2005)The natural course of non–classic Pompe’s disease; a review of 225 published cases J Neurol 252 875-884

Winkel LPF(2005)Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients Brain 128 671-677

Hagemans MLC(1999)Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling Eur J Hum Genet 7 713-716

Van Doorn PA(2011)Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy Orphanet J Rare Dis 6 34-1052

Loonen MCB(2011)Burden of illness of Pompe disease in patients only receiving supportive care J Inherit Metab Dis 34 1045-1692

Hop WJC(2004)Late-onset Pompe disease primarily affects quality of life in physical health domains Neurology 63 1688-1406

Reuser AJJ(2017)European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience Eur J Neurol 24 768-260

Hagemans MLC(2010)A randomized study of alglucosidase alfa in late-onset Pompe's disease N Engl J Med 362 1396-398

Winkel LPF(2016)Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up J Inherit Metab Dis 39 253-109