Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders

Prognostic Indicators of Renal Disease Progression in Adults with Fabry Disease: Natural History Data from the Fabry Registry

(2010) C. Wanner et al.   Clinical Journal of the American Society of Nephrology

The value of estimated GFR in comparison to measured GFR for the assessment of renal function in adult patients with Fabry disease

(2010) S. M. Rombach et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Disease Registries and Outcomes Research in Children

(2010) Simon Jones et al.   PEDIATRIC DRUGS

Force Majeure: Therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease

(2009) Carla E.M. Hollak et al.   BLOOD CELLS MOLECULES AND DISEASES

Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy

(2009) Frank Weidemann et al.   CIRCULATION

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1

(2009) Gregory A Grabowski et al.   GENETICS IN MEDICINE

Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey

(2009) A Mehta et al.   JOURNAL OF MEDICAL GENETICS

Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data

(2009) A Mehta et al.   LANCET

Vasculopathy in patients with Fabry disease: Current controversies and research directions

(2009) S.M. Rombach et al.   MOLECULAR GENETICS AND METABOLISM

Eight-Year Clinical Outcomes of Long-Term Enzyme Replacement Therapy for 884 Children With Gaucher Disease Type 1

(2008) H. Andersson et al.   PEDIATRICS

Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry

(2007) William R. Wilcox et al.   MOLECULAR GENETICS AND METABOLISM

Conditional approval: discussion points from the PSI conditional approval expert group

(2007) Kevin Carroll et al.   PHARMACEUTICAL STATISTICS