Infantile-onset or later-onset form of lysosomal acid lipase deficiency results in premature mortality in humans. Current treatment is denied approval in certain European countries due to its exorbitant cost. Our results indicate that anti-FGF19/FGFR4 treatment as combinational therapy might reduce the cost of disease management in affected patients.

Eingereicht von: Vinay Sachdev, PhD
Firma/Universität: Medical University of Graz
Homepage: www.medunigraz.at

The results of the study have great potential to be translated into patient care and for the use of individual subjects suffering from LAL-D. Currently, a recombinant enzyme replacement therapy for LAL-D is available. The results are promising with definite decrease in disease-related morbidity and mortality. However, given the extreme high cost of this treatment (~800,000€/patient/year) and lack of long-term treatment data in subjects, it is imperative to continue the search for adjunct therapies for patient welfare. In fact, the drug was denied U.K. approval due to its exorbitant cost. The societal impact of improved care of patients suffering from rare diseases is large, and the need for more effective combinational therapies is pressing. Our planned collaboration with prospective clinicians in Austria and abroad aims for early diagnosis and alleviation of the disease burden in LAL-D patients.

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