US 10,888,607 B2
Marker for acid sphingomyelinase disorders and uses thereof
Wei-Lien Chuang, Bridgewater, NJ (US); Gerald F. Cox, Bridgewater, NJ (US); and X. Kate Zhang, Bridgewater, NJ (US)
Assigned to Genzyme Corporation, Cambridge, MA (US)
Filed by Genzyme Corporation, Cambridge, MA (US)
Filed on Jun. 8, 2018, as Appl. No. 16/3,598.
Application 16/003,598 is a continuation of application No. 14/895,472, granted, now 10,022,428, previously published as PCT/US2014/041405, filed on Jun. 6, 2014.
Claims priority of provisional application 61/832,302, filed on Jun. 7, 2013.
Prior Publication US 2018/0289778 A1, Oct. 11, 2018
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 38/46 (2006.01); G01N 33/92 (2006.01)
CPC A61K 38/465 (2013.01) [G01N 33/92 (2013.01); C12Y 301/04012 (2013.01); G01N 2333/922 (2013.01); G01N 2405/08 (2013.01); G01N 2800/04 (2013.01); G01N 2800/52 (2013.01)] 15 Claims
 
1. A method of treating an acid sphingomyelinase (ASM) deficiency in a human subject, wherein the method comprises:
(a) administering to the subject a therapeutic agent for the ASM deficiency; and
(b) obtaining the level of lyso-sphingomyelin (lyso-SPM) in a biological sample from the subject collected three or more days after step (a), wherein the biological sample is a blood sample and a decrease of the level of lyso-SPM compared to a reference level indicates efficacy of the therapeutic agent.