Over the past three decades, enzyme therapy for lysosomal storage diseases has moved
from an academic pursuit to direct delivery of effective clinical care for affected patients and …

During the last few years, much progress has been made in the treatment of lysosomal
storage disorders. In the past, no specific therapy was available for the affected patients, and …

Background: The therapy of the lysosomal storage diseases (LSDs) was developed by
supplying adequate amounts of the needed enzyme to affected individuals. This approach in …

In the last years, much progress has been achieved in the field of lysosomal storage
disorders. In the past, no specific treatment was available for the affected patients; …

Potential treatment for lysosomal diseases now includes enzyme replacement therapy,
substrate reduction therapy, and chaperone therapy. The first two of these have been …

Targeted treatments for the lysosomal storage disorders (LSDs), in the form of enzyme
replacement and/or substrate depletion, have been shown to be relatively safe and effective …

Enzyme replacement therapy for the lysosomal storage disorders derives its impetus from
the successes achieved in the treatment of Gaucher disease. After nearly two decades of …

Abstract In 1882, Philippe Gaucher described a 32-year-old woman with massive
splenomegaly and unusually large cells in the spleen, which he called a “primary …

In 1964, Christian de Duve first suggested that enzyme replacement might prove therapeutic
for lysosomal storage diseases (LSDs). Early efforts identified the major obstacles, including …

The first enzyme replacement therapy (ERT) for a lysosomal storage disorder (LSD) was
approved in 1991 and we now have more than 25 years of experience of treating patients …