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Medicine & Law Weekly

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Addenbrookes Hospital, Cambridge



New data from Addenbrookes Hospital, Cambridge detailed



June 2nd, 2006

Researchers have identified novel biomarkers for Niemann-Pick disease using gene expression analysis of acid sphingomyelinase knockout mice.

According to a study from the United States, "Although several therapies are available or being developed for lysosomal storage disorders (LSDs), assessment of therapeutic efficacy is challenged by the lack of markers to assess disease progression and severity. This is particularly true for rare diseases such as LSDs, since natural history data from human populations are often lacking."

Rajwinder Dhami and colleagues at Mount Sinai School of Medicine and Genzyme Corporation wrote, "[In our study] we describe the...


Source: Medicine & Law Weekly (2006-06-02)

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