DODGE J C ET AL: "Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease", EXPERIMENTAL NEUROLOGY, ACADEMIC PRESS, NEW YORK, NY, US, vol. 215, no. 2, 1 February 2009 (2009-02-01), pages 349 - 357, XP025875199, ISSN: 0014-4886, [retrieved on 20090118], DOI: 10.1016/J.EXPNEUROL.2008.10.021 (B1)

MIRANDA SILVIA R P ET AL: "Infusion of recombinant human acid sphingomyelinase into Niemann-Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology", FASEB JOURNAL, vol. 14, no. 13, October 2000 (2000-10-01), pages 1988 - 1995, XP002691898, ISSN: 0892-6638 (B1)

ZIEGLER R J ET AL: "Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, SAN DIEGO, CA, US, vol. 97, no. 1, 1 May 2009 (2009-05-01), pages 35 - 42, XP026066935, ISSN: 1096-7192, [retrieved on 20090125], DOI: 10.1016/J.YMGME.2009.01.008 (B1)

SIMONARO CALOGERA M ET AL: "The demographics and distribution of type b Niemann-Pick disease: Novel mutations lead to new genotype/phenotype correlations.", AMERICAN JOURNAL OF HUMAN GENETICS, vol. 71, no. 6, December 2002 (2002-12-01), pages 1413 - 1419, XP002691899, ISSN: 0002-9297 (B1)

WO-A2-2007/084737 (B1)

PATAT A A: "Designing and interpreting the results of first-time-to-man studies.", DIALOGUES IN CLINICAL NEUROSCIENCE SEP 2000, vol. 2, no. 3, September 2000 (2000-09-01), pages 203 - 212, XP002691900, ISSN: 1294-8322 (B1)