Abstract: The present disclosure provides compositions and methods for treating amyotrophic lateral sclerosis (ALS). Among other things, the present disclosure provides inhibitory nucleic acids that inhibit the expression of genes that cause or are implicated in ALS pathogenesis. The present disclosure further provides recombinant adeno-associated virus (rAAV) vectors comprising inhibitory nucleic acids that inhibit the expression of genes that cause or are implicated in ALS pathogenesis.

Abstract: Featured are biornarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.

Abstract: Featured are biomarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.

Abstract: Aspects of the application relate to compositions and methods for treating spinal muscular atrophy in a subject. In particular, this application provides therapeutic combinations of a small molecule that promotes SMN function and/or a recombinant nucleic acid that encodes the survival of motor neuron 1 (SMN1) protein (e.g., in a viral vector), and/or an antisense oligonucleotide (ASO) that increases full-length survival of motor neuron 2 (SMN2) mRNA (e.g., that is targeted to a nucleic acid molecule encoding the survival of motor neuron 2 (SMN2) and promotes the inclusion of exon 7 in SMN2 mRNA).

Abstract: Aspects of the application relate to compositions and methods for treating spinal muscular atrophy in a subject. In particular, this application provides therapeutic combinations of a recombinant nucleic acid that encodes the survival of motor neuron 1 (SMN1) protein (e.g., in a viral vector), and an antisense oligonucleotide (ASO) that increases full-length survival of motor neuron 2 (SMN2) mRNA (e.g., that is targeted to a nucleic acid molecule encoding the survival of motor neuron 2 (SMN2) and that promotes the inclusion of exon 7 in SMN2 mRNA).

Abstract: Featured are biomarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.

Abstract: This invention is a method and kit for treating a disease associated with, or resulting from, the accumulation of soluble oligomer amyloid beta 1-42 using an antibody, or antibody fragment thereof, that has a higher affinity for amyloid beta 1-42 oligomers than for amyloid beta 1-42 monomer, amyloid beta 1-40 monomer, plaques and amyloid beta fibrils and, optionally, a tau therapeutic or an inhibitor of amyloid beta production or aggregation.

Abstract: This invention is a selective A? oligomer kit and immunoassay method capable of reliably and sensitively detecting A? oligomers in a biological sample of a patient. In one embodiment the inventive assay uses a pair of anti-A? oligomer antibodies, as capture and detection antibodies, to detect and quantify A? oligomers. The method can be used to differentiate Alzheimer's disease (AD) patients from non-AD patients and/or to stratify AD patients according to the severity of their disease.

Abstract: The present invention relates to antibodies that bind amyloid ?-derived diffusible ligands, also known as ADDLs. The antibodies of the invention are selective for ADDLs, can penetrate the brain, and are useful in methods of detecting ADDLs and diagnosing Alzheimer's disease. The present antibodies also block binding of ADDLs to neurons, assembly of ADDLS, and tau phosphorylation and are there useful in methods for the preventing and treating diseases associated with ADDLs.

Abstract: This invention is a selective A? oligomer kit and immunoassay method capable of reliably and sensitively detecting A? oligomers in a biological sample of a patient. In one embodiment the inventive assay uses a pair of anti-A? oligomer antibodies, as capture and detection antibodies, to detect and quantify A? oligomers. The method can be used to differentiate Alzheimer's disease (AD) patients from non-AD patients and/or to stratify AD patients according to the severity of their disease.

Abstract: This invention is a selective A? oligomer kit and immunoassay method capable of reliably and sensitively detecting A? oligomers in a biological sample of a patient. In one embodiment the inventive assay uses a pair of anti-A? oligomer antibodies, as capture and detection antibodies, to detect and quantify A? oligomers. The method can be used to differentiate Alzheimer's disease (AD) patients from non-AD patients and/or to stratify AD patients according to the severity of their disease.

Abstract: The present invention relates to antibodies that bind amyloid ?-derived diffusible ligands, also known as ADDLs. The antibodies of the invention are selective for ADDLs, can penetrate the brain, and are useful in methods of detecting ADDLs and diagnosing Alzheimer's disease. The present antibodies also block binding of ADDLs to neurons, assembly of ADDLS, and tau phosphorylation and are there useful in methods for the preventing and treating diseases associated with ADDLs.

Abstract: The invention herein is directed to a selective A? oligomer immunoassay capable of reliably and sensitively detecting A? oligomers in a biological sample of a patient. In one embodiment the inventive assay uses a pair of anti-A? oligomer antibodies, 19.3 and 82E1, to detect and quantify A? oligomers in a cerebrospinal fluid (CSF) sample. The inventive assay can be used to differentiate Alzheimer's disease (AD) patients from non-AD patients and/or to stratify AD patients according to the severity of their disease. The inventive assay can also be used as a target engagement assay that can measure bound A? oligomers as a surrogate end-point for the assessment of therapeutic efficacy and/or target engagement.

Abstract: The invention herein is directed to immunoassays for the detection of A?42 oligomers. The inventive assays are based on the observations herein that the presence of A?42 oligomers in a preparation is directly related to a decrease in a C-terminal (CT) immunosignal and a correlated increase in an N-terminal (NT) immunosignal, relative to the immunosignal generated in the absence of A?42 oligomers, in an A?42 CT and NT ELISA assay and an A?42 CT AlphaLISA assay. The invention herein involves the use of these assays alone or in combination to screen for inhibitors of A?42 oligomerization.

Abstract: Disclosed are antibodies that bind amyloid beta-derived diffusible ligands, also known as ADDLs. The antibodies are selective for ADDLs, can penetrate the brain, and are useful in methods of detecting ADDLs and diagnosing Alzheimer's disease. The antibodies also block binding of ADDLs to neurons, assembly of ADDLS, and tau phosphorylation and are there useful in methods for the preventing and treating diseases associated with ADDLs.

Abstract: The invention herein is directed to a selective A? oligomer immunoassay capable of reliably and sensitively detecting A? oligomers in a biological sample of a patient. In one embodiment the inventive assay uses a pair of anti-AP oligomer antibodies, 19.3 and 82E1, to detect and quantify A? oligomers in a cerebrospinal fluid (CSF) sample. The inventive assay can be used to differentiate Alzheimer's disease (AD) patients from non-AD patients and/or to stratify AD patients according to the severity of their disease. The inventive assay can also be used as a target engagement assay that can measure bound A? oligomers as a surrogate end-point for the assessment of therapeutic efficacy and/or target engagement.