The designation was based on data generated from clinical trials with sebelipase alfa in patients with early onset LAL Deficiency.
http://www.drugs.com/clinical_trials/synageva-s-sebelipase-alfa-receives-breakthrough-therapy-designation-15562.html?
http://www.drugs.com/clinical_trials/synageva-s-sebelipase-alfa-receives-breakthrough-therapy-designation-15562.html?
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