Abstract: The invention is based on the detection of microRNA (miR) in biological samples derived from subjects for assessing a risk that the subject develops a neurological and/or neuropsychological condition. The invention in particular detects the presence or absence and the amount of microRNA-181a-5p, microRNA146a-5p and/or microRNA148a-3p in such samples, which are shown by the invention to be associated with the neurological and/or neuropsychological condition. In addition, the invention provides a therapeutic application for the treatment of the neurological and/or neuropsychological condition by modulating the expression/function of these miR. Furthermore, the invention provides a method for monitoring the treatment success of the neurological and/or neuropsychological condition.

Abstract: The invention relates to a chimeric autoantibody receptor (CAAR) and nucleic acid molecules encoding said CAAR, wherein the CAAR comprises an extracellular domain comprising an autoantigen of a nicotinic acetylcholine receptor (nAChR) or fragment thereof. The invention relates further to a vector comprising a nucleic acid molecule encoding the CAAR, to a CAAR polypeptide, to a genetically modified cell expressing the CAAR or comprising a nucleic acid molecule or a vector encoding the CAAR. The invention relates further to genetically modified cells expressing the CAAR for use in the treatment of a neuromuscular disorder associated with autoantibodies that bind a nicotinic acetylcholine receptor (nAChR), preferably for the treatment of myasthenia gravis (MG).

Abstract: A modulator of PDE1A and/or PDE1C can be used as a medicament, in particular in the prevention or treatment of synucleinopathies, such as multiple system atrophy, dementia with Lewy bodies, Parkinson's disease, pure autonomic failure, rapid eye movement sleep behavior disorder, inherited synucleinopathies caused by mutations or multiplications of the SNCA gene, or synucleinopathies caused by mutations in other genes including, but not limited to, GBA, LRRK2 and PARK2.

Abstract: A modulator of PDE1A and/or PDE1C can be used as a medicament, in particular in the prevention or treatment of synucleinopathies, such as multiple system atrophy, dementia with Lewy bodies, Parkinson's disease, pure autonomic failure, rapid eye movement sleep behavior disorder, inherited synucleinopathies caused by mutations or multiplications of the SNCA gene, or synucleinopathies caused by mutations in other genes including, but not limited to, GBA, LRRK2 and PARK2.

Abstract: The present invention relates to a novel method for the treatment and/or prophylaxis of a tau-mediated neurodegenerative disease and/or of a tau-mediated neurodegenerative pathological condition, especially of a neurodegenerative disease and/or of a neurodegenerative pathological condition associated with and/or accompanied by tau aggregation, and in particular for the treatment and/or prophylaxis of a tauopathy; compounds and/or agents and compositions for such treatment and/or prophylaxis, and the manufacture of the compounds and/or agents and compositions suitable for the said treatment and/or prophylaxis. In this regard, the present invention relates especially to the use of compounds acting as PERK activator, a prodrug thereof, a derivative thereof and/or a pharmaceutically acceptable salt of any thereof, as a medicament.

Abstract: The present invention relates to a method of detecting a disease characterized by an expansion of genomic hexanucleotide repeats as well as polypeptides of said hexanucleotide repeats, ligands specifically binding to the polypeptides, and to methods of identifying an inhibitor preventing the expression and/or aggregation of said polypeptide.