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The National Collaborative Study for Lysosomal Storage Disorders (NCS-LSD)

In 2005, the Peninsula Medical School, in collaboration with the Specialist Treatment Centres for Lysosomal Storage Disorders in England, proposed to examine the effectiveness and associated costs of Enzyme Replacement Therapies for the treatment of LSDs. The Lysosomal Storage Disorder patient support groups worked closely alongside the study to provide advice on the collection of Quality of Life data.

The National Collaborative Study for Lysosomal Storage Disorders (NCS-LSD) collected clinical and Quality of Life information over three years, from consenting adults and children with Gaucher disease, Fabry disease, MPS I, MPS II, Pompe disease and Niemann Pick-C. The aim was to gain a better understanding of how effective these treatments are and what the associated costs are to society, and to the families who live with these conditions.

The full report is also available from the HTA website at:

In addition to the full report, a report has been sent to every family who participated in the study with a summary of the results relating to their condition, the Gaucher report is available to download click here.