Patent application number | Description | Published |
20110052559 | DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY - 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. | 03-03-2011 |
20130078230 | DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINOGMYELINASE DEFICIENCY - 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. | 03-28-2013 |
20130078231 | DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINOGMYELINASE DEFICIENCY - 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. | 03-28-2013 |
20130281511 | COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE - The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1. | 10-24-2013 |
20140199695 | Materials and Methods for Identifying Spinal Muscular Atrophy Carriers - Materials and methods for identifying carriers of genetic determinants of spinal muscular atrophy are disclosed. In particular, polymorphisms in linkage disequilibrium are associated as markers of spinal muscular atrophy alleles detectable by various techniques, including multiplex ligation-dependent probe analysis, sequence analysis, and RFLP detection. The materials and methods of the disclosure are particularly useful in identifying silent (2+0) carriers of spinal muscular atrophy in which two copies of the SMN1 gene are located on a single human chromosome 5 and no copies of the gene are located on the chromosome 5 homolog. | 07-17-2014 |
20140335070 | DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY - 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. | 11-13-2014 |
20150111841 | COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE - The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1. | 04-23-2015 |
20160115476 | COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE - The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1. | 04-28-2016 |