28 results about "Frontotemporal dementia" patented technology

The application relates to fused pyrimidinone derivatives of the general formula (I) which act as agonists of Triggering Receptor Expressed on Myeloid cells 2 (TREM2) useful for the treatment of Parkinson's disease, rheumatoid arthritis, Alzheimer's disease, amyotrophic lateral sclerosis, Nasu-Hakola disease, frontotemporal dementia, multiple sclerosis, prion disease, and stroke.

The invention relates to anti-TDP-43 binding molecules and uses thereof. The present invention is in the field of trans-activation responsive DNA binding proteins (TARDB or also referred to as TDP-43) having a molecular weight of 43 kDa. The present invention relates to TDP-43 specific binding molecules, in particular to anti-TDP-43 antibodies or antigen binding fragments or derivatives thereof, and uses thereof. The present invention provides means and methods for diagnosing, preventing, ameliorating and / or treating diseases, disorders and / or abnormalities associated with TDP-43 aggregates, including, but not limited to, frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), chronic traumatic encephalopathy (CTE), and edge-dominant age-related TDP-43 encephalopathy (LATE).

In various aspects of the present disclosure, there is provided a method of detecting, preventing, reversing, treating, or delaying the onset of a nervous system disease (e.g., an adult onset nervous system disease, Alzheimer's disease, Parkinson's disease, frontotemporal dementia).

The present disclosure relates to a compound of formula (I) which is in crystalline Form 1, characterized by having a powder X-ray diffractogram displaying peaks expressed as degree 2-Theta angles at about 3.3, 10.0, 20.1, 21.7, and 25.0. The present disclosure also relates to processes for its preparation, as well as a medicament and a pharmaceutical composition comprising it. The present disclosure further concerns the crystalline Form 1 of compound of formula (I) for use as a medicine and more particularly in the prevention and / or in the treatment of Parkinson's disease, frontotemporal dementia, multiple system atrophy, Alzheimer's disease, multiple sclerosis, amyotrophic lateral sclerosis, or brain injury.

The present disclosure relates to the novel use of ANAVEX3-71 and related compounds in medical treatments, such as Parkinson's Disease, Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease.

The present invention relates to novel compounds and pharmaceutical compositions. More particularly, the present invention provides novel compounds which are useful for the treatment of and prevention of neurodegenerative disorders including, but not limited to, Alzheimer's Disease (AD), Parkinson's Disease (PD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Nasu-Hakola Disease (NHD).

The present invention is in the field of trans-activation responsive DNA binding proteins (TARDB or also referred to as TDP-43) having a molecular weight of 43 kDa. The present invention relates to TDP-43 specific binding molecules, in particular to anti-TDP-43 antibodies or antigen binding fragments or derivatives thereof, and uses thereof. The present invention provides means and methods for diagnosing, preventing, ameliorating, and / or treating diseases, disorders, and / or abnormalities associated with TDP-43 aggregates, including, but not limited to, frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), chronic traumatic encephalopathy (CTE), and edge dominant age-related TDP-43 encephalopathy (LATE).

Provided herein are fusion proteins that comprise progranulin and an Fc polypeptide. Methods of using such proteins to treat progranulin-associated disorders (e.g., a neurodegenerative disease, such as frontotemporal dementia (FTD)) are also provided herein.

Disclosed are bicyclic heteroarene compounds, including purines, as PIKfyve inhibitors useful in the treatment of a TDP-43-associated neurological / neurodegenerative disorder, such as frontotemporal dementia, ALS and Alzheimer's disease. The compounds described herein, alone or in combination with other pharmaceutically active agents, can be used for treating or preventing such neurological / neurodegenerative diseases.

Among the various aspects of the present disclosure is the provision of a method of detecting, preventing, reversing, treating, or delaying the onset of a neurological disease (e.g., adult-onset neurological diseases, Alzheimer's disease, Parkinson's disease, Frontotemporal dementia).

The application relates to fused pyrimidinone derivatives of the general formula (I) which act as agonists of Triggering Receptor Expressed on Myeloid cells 2 (TREM2) useful for the treatment of Parkinson's disease, rheumatoid arthritis, Alzheimer's disease, amyotrophic lateral sclerosis, Nasu-Hakola disease, frontotemporal dementia, multiple sclerosis, prion disease, and stroke.

The present disclosure relates to a compound of formula (I) which is in crystalline Form 1, characterized by having a powder X-ray diffractogram displaying peaks expressed as degree 2-Theta angles at about 3.4, 6.8, 10.3, 13.7, and 20.5. The present disclosure also relates to processes for its preparation, as well as a medicament and a pharmaceutical composition comprising it. The present disclosure further concerns the crystalline Form 1 of compound of formula (I) for use as a medicine and more particularly in the prevention and / or in the treatment of Parkinson's disease, frontotemporal dementia, multiple system atrophy, Alzheimer's disease, multiple sclerosis, amyotrophic lateral sclerosis, or brain injury.

Disclosed herein are UNC13A oligonucleotides having one or more spacer regions or having no spacer regions. In various embodiments, UNC13A oligonucleotides with spacer regions can reduce misspliced UNC13A transcripts and increase full-length UNC13A transcripts, resulting in therapeutic efficacy on nervous system diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).

Disclosed are compounds of Formulas (I), (la), (lb), (II), (Ila), (III), (Illa), and (Illb), as well as pharmaceutical compositions thereof. The compounds can be used to improve proteostasis and enhance clearance of protein accumulation events by positively modulating the autophagy-lysosomal pathway, including augmenting the activity of cathepsin enzymes, and / or to treat neurological diseases, disorders and conditions, such as, but not limited to, Alzheimer's disease, Parkinson's disease, Huntington's disease, mild cognitive impairment, frontotemporal dementia, amyotrophic lateral sclerosis, Lewy body dementias, chronic traumatic encephalopathy, traumatic brain injury, and α-synucleinopathies.

Among the various aspects of the present disclosure is the provision of a method of detecting, preventing, reversing, treating, or delaying the onset of a neurological disease (e.g., adult-onset neurological diseases, Alzheimers disease, Parkinsons disease, Frontotemporal dementia).

Disclosed are compounds of Formulas (I), (Ia), (Ib), (II), (IIa), (III), (IIIa), and (IIIb), as well as pharmaceutical compositions thereof. The compounds can be used to improve proteostasis and enhance clearance of protein accumulation events by positively modulating the autophagy-lysosomal pathway, including augmenting the activity of cathepsin enzymes, and / or to treat neurological diseases, disorders and conditions, such as, but not limited to, Alzheimer's disease, Parkinson's disease, Huntington's disease, mild cognitive impairment, frontotemporal dementia, amyotrophic lateral sclerosis, Lewy body dementias, chronic traumatic encephalopathy, traumatic brain injury, and α-synucleinopathies.

The present invention provides a formulation of metformin and glibenclamide for the prevention and / or treatment of neurodegenerative diseases, preferably chosen among Parkinson's disease, multiple system atrophy, amyotrophic lateral sclerosis, frontotemporal dementia, Lewy body dementia, Huntington's disease and Alzheimer's disease.

Aspects of the disclosure relate to methods and compositions (e.g., kits) for detecting repeat-associated non-ATG (RAN) proteins in a subject (e.g., a subject having or suspected of having a disease associated with RAN protein translation, for example amyotrophic lateral sclerosis (ALS) and / or frontotemporal dementia (FTD), or spinocerebellar ataxia type 36 (SCA36), or other diseases that produce poly(PR), poly(GR) or poly(GP) RAN proteins. In some embodiments, methods described by the disclosure comprise detecting one or more RAN proteins in a biological sample obtained from a subject by an electrochemiluminescence-based immunoassay using one or more anti-RAN protein antibodies. In some embodiments, the disclosure relates to kits comprising one or more anti-RAN antibodies and an electrochemiluminescence-based immunoassay plate and / or reagents.

The present disclosure describes that the 15-lipoxygenase (15-LO) product 4-hydroxynonenal (4HNE or 4-HNE) post-translationally modifies LRRK2 and is believed to induce a pathologically elevated, chronic LRRK2 kinase activity associated with a LRRK2 mediated disease or condition, e.g., Parkinson's disease or frontotemporal dementia. Production of LRRK2-4HNE adducts, e.g., LRRK2 Cys2024-4HNE and / or LRRK2 Cys2025-4HNE adduct described herein, may be an indication of a LRRK2 mediated disease or condition. Inhibiting the production of excessive 4HNE in cells by 15-LO inhibitors converted an elevated LRRK2 activity to a normal basal LRRK2 activity level as demonstrated in cellular experiments in the Examples. The 15-LO inhibition of 4HNE production is believed to only remove elevated LRRK2 activity associated with LRRK2 mediated diseases or conditions and not normal LRRK2 activity that may be required for other cellular functions. Accordingly, 15-LO inhibition of elevated LRRK2 activity and / or expression may afford a safe and effective treatment for LRRK2 mediated diseases or conditions.

Provided is a fusion protein comprising an anti-CD98 antibody fused to a human progranulin (PGRN) polypeptide. Uses in the treatment of neurodegenerative diseases such as frontotemporal dementia (FTD) and lysosomal storage disorders are also provided.

Disclosed herein are UNC13A antisense oligonucleotides with modified backbone structures. The disclosed UNC13A oligonucleotides reduce mis-spliced UNC13A transcripts and increase full length UNC13A transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).

The present invention relates to anti-sortilin monoclonal antibodies that have been found to be useful in correcting a deficient level of progranulin (PGRN). In particular, these antibodies can be used in the treatment of frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), and other neurodegenerative disorders, such as Alzheimer's disease (AD).

The present disclosure provides for a compound according to formula (I)or a pharmaceutically acceptable salt thereof as CaV1.22 activators for the treatment of schizophrenia, bipolar disorder, major depressive disorder, substance use disorder, ADHD, Phelan-McDermid Syndrome, autism spectrum disorder, multiple sclerosis, frontotemporal dementia, Alzheimer's disease, Brugada Syndrome, Short QT syndrome, or early repolarization syndrome.

The present invention discloses compositions, means and kits thereof for treating neuronal clinical indications in a mammalian subject. The composition comprises, inter alia, a synergistic combination of an anti-inflammatory drug and a DICER activator. The present invention further discloses methods for treating neuronal diseases including Motor neuron diseases (MNDs), ALS, FTD (Frontotemporal Dementia), macular degeneration (AMD) autism, and neurodegenerative diseases such as Parkinson's disease and Alzheimer's disease.

The present invention relates to the use of HIP / PAP proteins or derivatives thereof in therapy and prophylaxis, in particular to the use of said HIP / PAP proteins or derivatives thereof in the treatment of cognitive disorders associated with one or more anxiety disorders in an individual in need thereof, and for improving cognitive function in an individual affected by a nervous system condition associated with one or more anxiety disorders, or for alleviating cognitive deficits in a subject suffering from a condition selected from the group consisting of obsessive-compulsive disorder, attention deficit disorder, Lewy body dementia, early onset dementia, epilepsy-related cognitive dysfunction, frontotemporal dementia, posterior cortical atrophy, Huntington's disease (HD), Parkinson's disease, bipolar affective disorder, substance abuse, attention deficit disorder, etc. A psychiatric disorder and an SARS-CoV-2 infection; and for the prevention and / or treatment of diet-induced cognitive and anxiety deficits in an individual in need thereof, in particular for the prevention and / or treatment of cognitive and anxiety deficits caused by high fat diet.

The present invention relates to methods of treating diseases or disorders that are mediated by the loss of synaptic connectivity and / or plasticity, such as neurological diseases and disorders including obsessive compulsive disorder (OCD), Alzheimer's disease psychosis or psychosis in Alzheimer's disease (AD-P), frontotemporal dementia (FID), behavioral and psychological symptoms in dementia, motor symptoms in Parkinson's disease, and behavioral and psychological symptoms in Parkinson's disease, with (R)-1- (5-methoxy-1H-indol-1-y1)-N,N-dimethylpropan-2-amine (zalsupindole; AAZ-A-154). or a pharmaceutically acceptable salt or solvate thereof.

Composition and methods for treating neurodegenerative diseases, such as Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Angelman's Syndrome (AS), the compositions specifically including antisense oligonucleotides (ASOs) containing thiomorpholino nucleotides configured to inhibit ribosomal frameshifting of paternally expressed gene 10 (PEG 10) mRNA during translation, thereby inhibiting the formation of the long form gag-pol protein.