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US 10,213,494 B2Granted
Treatment of synucleinopathies
AssigneesThe Brigham and Women's Hospital, Inc., Genzyme Corporation
InventorsMichael Schlossmacher, Lamya Shihabuddin, Seng H. Cheng, Valerie Cullen
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Abstract
This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
Claims8
A method of treating a subject with a synucleinopathy, but not a clinically diagnosed lysosomal storage disease, wherein the synucleinopathy comprises any one or more of:
- sporadic or heritable dementia with Lewy bodies (DLB);
- pure autonomic failure (PAF) with α-synuclein deposition;
- multiple system atrophy (MSA);
- hereditary neurodegeneration with brain iron accumulation;
- incidental Lewy body disease of advanced age;
- Down's syndrome;
- progressive supranuclear palsy;
- essential tremor with Lewy bodies;
- tau gene and progranulin gene-linked dementia without parkinsonism;
- Creutzfeldt Jakob disease;
- bovine spongiform encephalopathy;
- sporadic or heritable spinocerebellar ataxia;
- and idiopathic rapid eye movement sleep behavior disorder, the method comprising administering to a subject any one or more of:
- a prosaposin polypeptide;
- a polynucleotide encoding a prosaposin polypeptide;
- a saposin A, B, or D polypeptide;
- and
- a polynucleotide encoding a saposin A, B, or D polypeptide;
- in an amount effective to reduce a level of a-synuclein in the subject's nervous system or in the subject's lysosomal compartment.
16 more elements
Dependencies are inferred from the claim text.