| 摘要 |
<p>The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.</p> |
| 申请人 |
MOUNT SINAI SCHOOL OF MEDICINE OF NEW YORK UNIVERSITY;GENZYME CORPORATION;SCHUCHMAN, EDWARD, H.;DESNICK, ROBERT, J.;COX, GERALD, F.;ANDREWS, LAURA, P.;MURRAY, JAMES, M. |
发明人 |
SCHUCHMAN, EDWARD, H.;DESNICK, ROBERT, J.;COX, GERALD, F.;ANDREWS, LAURA, P.;MURRAY, JAMES, M. |
