57 results about "Frontotemporal dementia" patented technology

Novel compounds such as DHMP maintain DHM features and pharmacological activities with increased potency and efficacy, improved water solubility, enhanced bioavailability, increased permeability to the blood brain barrier (BBB), and increased stability. Because DHMP retains the therapeutic activities of DHM and has properties that improve its bioavailability, it can be used as a therapeutic agent for treating Alzheimer's disease-related diseases (ADRD) including Alzheimer's disease (AD), Vascular Dementia, Frontotemporal Dementia (FTD), Mixed Dementia as well as neurodegeneration such as Parkinson's disease. Administration of these compounds result in beneficial pathological changes including reduction of Aꞵ and Tau, improvement of neuroinflammation, reduction of cognitive / memory deficits, as well as improvement of anxiety, depression, and aggression. The disclosed DHM derivatives can also be used to treat sleep disorders, neurological disorders such as PTSD, Autism, Epilepsy / Seizures, and Alcohol Use Disorders (AUD), fetal alcohol syndrome (FAD), substance addictions, and cancers.

The present invention is in the field of transactive response DNA binding protein with a molecular weight of 43 kDa (TARDB or also TDP-43). The invention relates to humanized TDP-43 specific binding molecules, in particular to humanized anti-TDP-43 antibodies or antigen-binding fragment or a derivative thereof and uses thereof. The present invention provides means and methods to diagnose, prevent, alleviate and / or treat a disease, disorder and / or abnormality 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 limbic-predominant age-related TDP-43 encephalopathy (LATE).

Disclosed are compounds, and corresponding methods of treatment, that activate myocyte-specific enhancer factor 2 (MEF2) transcriptional activity that are therefore useful in treating deficits in MEF2C activity found in autism spectrum disorder (ASD), intellectual disability (ID), attention deficit and hyperactivity disorder (ADHD), and in diseases characterized by cognitive decline such as Alzheimer's disease (AD), Lewy body dementia (LED), Frontotemporal dementia (FTD), and other forms of dementia, as well as movement disorders such as Parkinson's disease (PD) and parkinsonism from other causes.

Examples may provide a method of determining a neurological disorder status of a subject. The method includes generating at least one dynamic brain network model (DNM) for the subject, and determining at least one sink index (SI) for the subject using the DNM. The method also includes determining whether the subject has frontotemporal dementia (FTD), Alzheimer's disease, or does not have dementia using one or more SI ratios of brain regions associated with FTD to other brain regions. Additional methods as well as related systems and computer readable media are also provided.

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).

Methods and compositions for the treatment of cognitive disorders are provided herein.The present disclosure generally relates to the field of methods for delivering gene therapy to the entorhinal / hippocampal region accurately and safely to treat cognitive disorders and other diseases of the central nervous system (CNS).The present disclosure describes the specific parameters for targeting this network of brain regions, including accurate anatomical target, vector concentration and vector volume, and the subject may be at risk of suffering from a condition selected from Alzheimer's disease (AD), mild cognitive impairment, pre-symptomatic AD, frontotemporal dementia, or Lewy body 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.

Pyrvinium compounds, or a pharmaceutically acceptable salt thereof, used in methods for reducing tauopathy in a subject in need thereof. Pyrvinium compounds, or a pharmaceutically acceptable salt thereof, used in methods for treating a disease associated with accumulation of misfolded proteins (e.g., proteinopathies, Alzheimer's disease, frontotemporal dementia, Parkinson's disease, retinitis pigmentosa such as retinitis pigmentosa with rhodopsin mutations, aging, neurodegenerative diseases, or metabolic disorders).

The present invention relates to antisense oligonucleotides capable of modulating the expression of Tau in a target cell. The oligonucleotide lines hybridize to the MAPT mRNA. The present invention further relates to conjugates of the oligonucleotides, and pharmaceutical compositions and methods for the treatment of a Tau aberrant deposition lesion (Tauopathy), Alzheimer's disease (AD), frontotemporal dementia (FTD), FTDP-17, progressive ocular nuclear paralysis (PSP), chronic traumatic brain lesion (CTE), corticobasal nuclear degeneration (CBD), epilepsy, a Display syndrome, depression, a seizure disorder (disorder of epileptic seizure), and a movement disorder (disorder of movement).

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 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 belongs to the field of transactivation-responsive DNA binding protein with a molecular weight of 43 kDa (TARDB or also known as TDP-43). The present invention relates to TDP-43 specific binding molecules, in particular anti-TDP-43 antibodies or antigen-binding fragments or derivatives thereof and their uses. The present invention provides means and methods for diagnosing, preventing, alleviating 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 limbic-dominant age-related TDP-43 encephalopathy (LATE).

The present invention provides a method of treating frontotemporal dementia, or a childhood genetic neurodegenerative disease such as Ataxia Telangiectasia (A-T), or neurodegenerative diseases such as Parkinson's disease or neuropsychiatric diseases comprising administering to a subject in need thereof an effective amount of chlorite composition, such as sodium chlorite. The present invention thereby provides a method of modulating the immune system in a subject in need thereof. Described herein are methods of administration and treatment.

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.

The present disclosure provides human Tau protein (Tau)-binding proteins comprising an antigen binding domain of an antibody, wherein the antigen binding domain binds specifically to an epitope of Tau that results in inhibition of Tau aggregate seeding, and could be used to inhibit spreading of Tau aggregates in vivo. Also disclosed herein are nucleic acids, expression vectors, and recombinant viruses encoding such Tau-binding proteins, as well as compositions comprising such Tau-binding proteins. Also disclosed are methods for use of such compositions in the diagnosis, prophylaxis, treatment, and prognosis of Tauopathies such as Alzheimer's Disease, frontotemporal dementia, Pick's disease, and progressive supranuclear palsy. Genetically modified cells for expression for the disclosed Tau-binding proteins, and their use for production of the Tau-binding proteins are also disclosed.

There is described a set of antisense oligonucleotides suitable for inducing exon skipping on a pre-mature RNA transcript (pre-mRNA) from a human Granulin gene (GRN) carrying a null mutation. There are further described a pharmaceutical composition comprising said set of antisense oligonucleotides and the therapeutic use of said oligo set and / or pharmaceutical composition, particularly for the prevention and / or therapeutic treatment of a neurodegenerative disease such as frontotemporal dementia (FTD).

The present disclosure relates, in some aspects, to compositions of UNC13A antisense oligonucleotides (ASOs) and methods of using ASO to alter UNC13A concealment exon splicing and increase UNC13A protein expression. Such ASO may be used in the treatment of neurodegenerative disorders, in particular neurodegenerative disorders associated with the TAR-DNA binding protein-43 (TDP-43) pathology, such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).

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 against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).

Provided are a fused bicyclic heteroaryl amide compound as a protein aggregation inhibitor, as well as the use of such compound in the treatment or prevention of neurodegenerative diseases characterized by protein aggregation, such as Alzheimer's disease, Parkinson's disease, frontotemporal dementia, dementia with Lewy bodies, Parkinson's disease dementia, multiple system atrophy, amyotrophic lateral sclerosis, Huntington's disease, and cancer.

Described herein are compounds and methods for tethering proteins. For example, dimers of Protein X listed in Table 1 are described, where the dimers are formed by the covalent bonding of a cysteine on the first monomer to a cysteine on the second monomer via a cyclic disulfide linker. The covalently attached dimers exhibit increased stabilization and can be used to treat neurodegenerative diseases (such as, for example, Parkinson's Disease, ALS, Alzheimer's Disease, Huntington's Disease, Epilepsy, Frontotemporal Dementia, and / or DMD), cancer, autoimmune disease, and / or Celiac disease.

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).