amyotrophic

Tauroursodeoxycholate (UR 906), also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

Riluzole (RP-54274) is a glutamate antagonist (RECEPTORS, GLUTAMATE) used as an anticonvulsant (ANTICONVULSANTS) and to prolong the survival of patients with AMYOTROPHIC LATERAL SCLEROSIS.

(2R,3S)-N-(4-(2,6-dimethoxyphenyl)-5-(5-methylpyridin-3-yl)-4H-1,2,4-triazol-3-yl)-3-(5-methylpyrimidin-2-yl)butane-2-sulfonamide is an Apelin receptor agonist with an EC50 for Apelin receptors of 0.012 µM.(2R,3S)-N-(4-(2,6-dimethoxyphenyl)-5-(5-methylpyridin-3-yl)-4H-1,2,4-triazol-3-yl)-3-(5- methylpyrimidin-2-yl)butane-2-sulfonamide is used in the treatment of heart failure, atherosclerosis, obesity, diabetes mellitus, amyotrophic lateral sclerosis, coronary artery disease, hypertension, stroke and myocardial infarction.

Arimoclomol (BRX-220 free base), a co-inducer of heat shock proteins (HSP), is utilized in studies on the treatment of amyotrophic lateral sclerosis (ALS).

DNL343 is a selective and potent eIF2B activator that crosses the blood-brain barrier and inhibits the assembly of stress granules (SGs) induced by the C9ORF72 dipeptide repeats. DNL343 may be useful in the study of neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS).

Utreloxastat (PTC857) is a novel 15-lipoxygenase inhibitor that can be used to study amyotrophic lateral sclerosis .

MTP 131 is a mitochondria-targeted peptide antioxidant.1,2It localizes to the mitochondria and reducestert-butyl hydroperoxide-induced lipid peroxidation and apoptosis in SH-SY5Y cells when used at concentrations ranging from 0.001 to 1 nM.1MTP 131 (2 mg kg) reduces infarct volume, hemispheric swelling, and glutathione (GSH) depletion in a mouse model of acute cerebral ischemia induced by middle cerebral artery occlusion (MCAO).2It increases survival, improves motor function, and decreases degeneration of the lumbar spinal cord in a superoxide dismutase 1 mutant (SOD1G93A) transgenic mouse model of amyotrophic lateral sclerosis (ALS) when administered at a dose of 5 mg kg. MTP 131 reduces albuminuria, urinary hydrogen peroxide levels, and mesangial matrix accumulation, as well as preserves superoxide production, in adb dbmouse model of diabetic nephropathy.3 1.Zhao, K., Luo, G., Giannelli, S., et al.Mitochondria-targeted peptide prevents mitochondrial depolarization and apoptosis induced by tert-butyl hydroperoxide in neuronal cell linesBiochem. Pharmacol.70(12)1796-1806(2005) 2.Szeto, H.H.Mitochondria-targeted peptide antioxidants: Novel neuroprotective agentsAAPS J.8(3)E521-E531(2006) 3.Miyamoto, S., Zhang, G., Hall, D., et al.Restoring mitochondrial superoxide levels with elamipretide (MTP-131) protects db db mice against progression of diabetic kidney diseaseJ. Biol. Chem.295(21)7249-7260(2020)

SOD1-Derlin-1 inhibitor 56-59 is an inhibitor of SOD1-Derlin-1 interaction. SOD1-Derlin-1 inhibitor 56-59 is potential in Amyotrophic lateral sclerosis (ALS) research.

NF-κΒ Activator 2 is a potent, orally active compound that efficiently activates NF-κB with an EC50 value of 1.58 μM. By enhancing NF-κB expression and activation, it induces SOD 2 synthesis, rendering it useful for amyotrophic lateral sclerosis (ALS) research [1].

Tauroursodeoxycholic-2,2,3,4,4-d5 Acid is a deuterated compound of Tauroursodeoxycholic Acid. Tauroursodeoxycholic Acid has a CAS number of 14605-22-2. Tauroursodeoxycholate, also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

Tauroursodeoxycholic Acid-d4 Sodium Salt is a deuterated compound of Tauroursodeoxycholic Acid Sodium Salt. Tauroursodeoxycholic Acid Sodium Salt has a CAS number of 14605-22-2. Tauroursodeoxycholate, also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

ROCK2-IN-5, a compound integrating structural elements from the Rho kinase inhibitor fasudil and the NRF2 inducers caffeic and ferulic acids, exhibits a favorable multitarget profile and tolerability. It shows potential for research in Amyotrophic Lateral Sclerosis (ALS) associated with a SOD1 mutation.

SOD1-Derlin-1 inhibitor-1 (SOD1-Derlin-1 Inhibitor 56-20) (compound 56-20) is an inhibitor of SOD1-Derlin-1 interaction. SOD1-Derlin-1 inhibitor-1 inhibits SOD1 G93A -Derlin-1 complex with an IC 50 value of 7.11 μM. SOD1-Derlin-1 inhibitor-1 can be used for amyotrophic lateral sclerosis research[1].

CMB-087229 is an inhibitor of mutant superoxide dismutase 1 (SOD1) protein aggregation, exhibiting an IC50 of 67 nM, and holds potential for use in amyotrophic lateral sclerosis research [1].

Chrexanthomycin C, an orally active marine-derived natural product, exhibits significant bioactivities and shows a binding affinity for DNA [(G4C2)4 G4] with a dissociation constant (Kd) of 2.8 mM. This compound holds potential for neurodegenerative disease research, including amyotrophic lateral sclerosis (ALS) [1].

K812 is an ASK1-specific inhibitor discovered to prolong survival in a mouse model of amyotrophic lateral sclerosis.

c-ABL-IN-3, a highly effective c-Abl inhibitor, exhibits promising potential for studying neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and Parkinson's disease (PD), as well as cancer [1]. The activation of c-Abl has been strongly implicated in a range of diseases, notably cancer [1]. It offers valuable opportunities for research in these fields [1].

BSc5367 is a potent inhibitor for the Nek1 kinase domain with an IC50 value of 11.5 nM. Nek1 is a NIMA-related protein kinase that crucially involved in cell cycle regulation, DNA repair and microtubule regulation. The Nek1 dysfunctions play key roles in amyotrophic lateral sclerosis (ALS), polycystic kidney disease (PKD) and several types of radiotherapy resistant cancer. BSc5367 provides a useful tool to better understand the aforementioned diseases[1].

The metallo-protein Cu/Zn-superoxide dismutase (SOD1) is a ubiquitous enzyme responsible for scavenging superoxide radicals. Mutations in SOD1, which alter its metal binding capacity and can result in protein misfolding and aggregation, have been linked to familial amyotrophic lateral sclerosis (ALS). Cu-ATSM is an orally bioavailable, blood-brain barrier permeable complex that has traditionally been used in cellular imaging experiments to selectively label hypoxic tissue via its susceptibility to reduction by oxygen-depleted mitochondria. More recently, Cu-ATSM has been reported to improve locomotor function and survival in a transgenic ALS mouse model by delivering copper specifically to cells in the spinal cords of mice producing misfolded SOD1 proteins. Copper chaperone for SOD (CCS) is presumed to utilize the copper from Cu-ATSM to prevent misfolding of the SOD1 protein.

Atibuclimab is a chimeric monoclonal antibody targeting CD14, consisting of a mouse variable region and a human IgG4 Fc region. Atibuclimab can be used to treat amyotrophic lateral sclerosis. Atibuclimab reduces LPS-induced disease and inhibits LPS-induced pro-inflammatory cytokine release, delaying the release of anti-inflammatory cytokine soluble TNF receptor type I.

Di-Ellipticine-RIBOTAC is a bifunctional small molecule that effectively diminishes the r(G4C2) repeat expansion associated with c9ALS/FTD both in vitro and in vivo, showcasing therapeutic potential in amyotrophic lateral sclerosis (ALS) models.

EPI-589, a quinone derivative and oxidoreductase enzyme inhibitor, is considered safe and well tolerated, showing promise for the treatment of amyotrophic lateral sclerosis (ALS).

Riluzole-13C,15N2 is intended for use as an internal standard for the quantification of riluzole by GC- or LC-MS. Riluzole is a benzothiazole derivative with anti-excitotoxic effects that acts by blocking the presynaptic release of glutamate, indirectly antagonizing glutamate receptors, and inactivating neuronal voltage-gated Na+ channels (ED50 = 2.3 μM). Riluzole suppresses glutamate-induced seizures in rats at an ED50 value of 3.2 mg/kg and displays neuroprotective effects in hypoxic animals at an ED50 value of 4 mg/kg. Formulations containing riluzole have been explored as therapeutics for slowing disease progression of amyotrophic lateral sclerosis.

Tauroursodeoxycholic-d4 Acid is a deuterated compound of Tauroursodeoxycholic Acid. Tauroursodeoxycholic Acid has a CAS number of 14605-22-2. Tauroursodeoxycholate, also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

UCPH-102 can be used in Alzheimer’s disease, amyotrophic lateral sclerosis, chronic pain and obsessive compulsive disorder studies with good blood-brain permeability. UCPH-102 is a highly selective inhibitor of EAAT1 with an IC50 value of 0.43 μM. UCPH-102 also shows a specific anti-proliferative effect on T-ALL cells [1] [2] [3] [4].

GM-604(alirinetide) is a potential treatment for amyotrophic lateral sclerosis and ischemic stroke.

Proteasome-activating peptide 1 enhances chymotrypsin-like proteasomal catalytic activity, boosting proteolytic rates both in vitro and within cultured cells. Additionally, it inhibits protein aggregation in a cellular model of amyotrophic lateral sclerosis [1].

Ozanezumab (GSK1223249) is a monoclonal antibody targeting Nogo-A (neurite outgrowth inhibitor A). It is utilized in research related to amyotrophic lateral sclerosis (ALS) and multiple sclerosis [1].

Apilimod 2HCl (STA 5326 2HCl) is a PIKFYVE kinase inhibitor that promotes NLRP3 inflammatory vesicle activation, IL-1β secretion, and cellular pyroptosis, and is used in the study of amyotrophic lateral sclerosis.

Sotuletinib (BLZ945) dihydrochloride is an orally administered, blood-brain barrier-permeable CSF1-R inhibitor with an IC50 of 1 nM, promoting apoptosis in tumor cells and significantly restricting tumor growth in murine models, thereby proving useful in cancer and amyotrophic lateral sclerosis (ALS) studies [1].

WN1316 is a unique neuroprotectant against oxidative injury, being a highly promising remedy for the treatment of amyotrophic lateral sclerosis (ALS).

β-N-Methylamino-L-alanine hydrochloride (L-BMAA hydrochloride) is a neurotoxin produced by cyanobacteria that may contribute to the development of amyotrophic lateral sclerosis (ALS) and possibly other neurodegenerative diseases.

C-ABL-IN-2 is a highly effective inhibitor of the c-Abl protein, which plays a significant role in the development of several diseases, including cancer. This compound exhibits promise for investigating neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS) and Parkinson's disease (PD), as well as cancer. (Source: WO2020260871A1, compound 25) [1].

PDGFR Tyrosine Kinase Inhibitor III (PDGF Receptor Tyrosine Kinase Inhibitor III) is a multikinase inhibitor that inhibits PDGFR, EGFR, FGFR, PKA, and PKC and can be used for research on amyotrophic lateral sclerosis [1].

Proteasome-activating peptide 1 TFA is a potent activator of the proteasome, enhancing chymotrypsin-like proteasomal catalytic activity and increasing proteolytic rates both in vitro and in culture. It also inhibits protein aggregation in cellular models of amyotrophic lateral sclerosis (ALS) [1].

Riluzole hydrochloride (PK 26124 hydrochloride) is an anticonvulsant compound belonging to the family of sodium channel blockers that inhibits GABA uptake.Riluzole hydrochloride is a sodium-glutamate antagonist with anticancer and neuroprotective activity and can be used to study amyotrophic lateral sclerosis and neurological disorders.

Tofersen (BIIB067) is an antisense oligonucleotide used to inhibit the synthesis of superoxide dismutase 1 (SOD1) protein by facilitating the degradation of SOD1 mRNA via an RNase H-dependent mechanism. This compound holds potential for amyotrophic lateral sclerosis (ALS) research.

1-Stearoyl-2-docosahexaenoyl-sn-glycero-3-PC is a phospholipid in biological membranes containing stearic acid and docosahexaenoic acid at the sn-1 and sn-2 positions, respectively. It has been used to study the organization and dynamics of lipid membranes. In the superoxide dismutase 1 mutant transgenic mouse model of amyotrophic lateral sclerosis (ALS) 1-Stearoyl-2-docosahexaenoyl-sn-glycero-3-PC levels decrease in the anterior horn of L5 in late stages of the disease.1-Stearoyl-2-docosahexaenoyl-sn-glycero-3-PC can be used to study biological membranes.

K811 is an ASK1-specific inhibitor that prolongs survival in a mouse model of amyotrophic lateral sclerosis. K811 efficiently prevented cell proliferation in cell lines with high ASK1 expression and in HER2-overexpressing GC cells. Treatment with K811 reduced sizes of xenograft tumors by downregulating proliferation markers.

Ulefnersen, a compound that reduces the synthesis of the RNA-binding protein fused-in sarcoma (FUS), has applications in Amyotrophic Lateral Sclerosis (ALS) research.

NUCC-0000323 is a potent inhibitor of superoxide dismutase 1 (SOD1), effectively suppressing SOD1 expression. This compound has applications in amyotrophic lateral sclerosis (ALS) research.