VIRAL GENE THERAPY AS TREATMENT FOR CHOLESTEROL STORAGE DISEASE OR DISORDER
US Application 62/144,702
Filed on 2015-04-08
Filed on 2015-04-08
METHODS OF TREATING PXE WITH TNAP INHIBITORS
US Application 62/058,505
Filed on 2014-10-01
Filed on 2014-10-01
Compounds and Method for Treating PARP1-Deficient Cancers
US Application 61/988,502
Filed on 2014-05-05
Filed on 2014-05-05
Compounds and Method for Treating PARP1-Deficient Cancers
US Application 61/930,291
Filed on 2014-01-22
Filed on 2014-01-22
METHODS FOR TREATING LEUKEMIA AND DISORDERS MEDIATED BY MLL FUSION PROTEINS
US Application 61/736,459
Filed on 2012-12-12
Filed on 2012-12-12
Compositions and Methods for Predicting Age of Onset of A Lysosomal Storage Disease or a Disease Associated with a Lysosomal Defect
US Application 61/781,807
Filed on 2013-03-14
Filed on 2013-03-14
Biomarker For Hyposialylation Disorders
US Application 61/785,094
Filed on 2013-03-14
Filed on 2013-03-14
Methods For Treating Progeroid Laminopathies Using Oligonucleotide Analogues Targeting Human Lamin A (LMNA)
US Application 61/568,590
Filed on 2011-12-08
Filed on 2011-12-08
Synthetic Methylmalonyl-CoA Mutase Transgene For The Treatment Of Mut Class Methylmalonic Acidemia (MMA)
US Application 61/792,081
Filed on 2013-03-15
Filed on 2013-03-15