progressive

BAF312 (Siponimod (BAF-312)), a next-generation S1P receptor modulator, is specific for S1P1 and S1P5 receptors with EC50 of 0.39 nM and 0.98 nM, respectively. The specificity of BAF312 for S1P1 and S1P5 receptors exhibits >1000-fold over S1P2, S1P3 and S1P4 receptors.

LDN193189 (LDN-193189) (DM 3189) is a selective BMP signaling inhibitor, inhibiting the transcriptional activity of ALK2 and ALK3 (IC50s: 0.8 0.8 5.3 16.7 nM for ALK1 2 3 6).

UCM-05194 is a selective LPA1 receptor agonist that can improve neuropathic pain and can be used in the study of progressive system diseases.

PZL-A, an activator of mtDNA synthesis, restored the function and activity of the most common POLγ mutant variant and activated mtDNA synthesis in the cells of pediatric patients with fatal diseases, thereby enhancing the oxidative phosphorylation mechanism and the biogenesis of cellular respiration, and can be used to treat progressive diseases associated with mtDNA depletion.

LDN 193688 is a BMP kinase inhibitor preferential for the ALK2 site, acting by binding to the ALK site, with BMP inhibitory activity 100 times that of TGFβRI, used in the study of progressive osseous fibrodysplasia.

HDAC6-IN-52 (EX.1) is a potent HDAC6 inhibitor, achieving a 100% inhibition rate at a concentration of 10 μM. It plays a significant role in central nervous system disorders, including neurodegenerative diseases such as Alzheimer's disease and progressive supranuclear palsy.

Tau ligand-1 (Compound 75) is a ligand of aggregated tau protein that can penetrate the blood-brain barrier. In tissues affected by Alzheimer's disease, progressive supranuclear palsy, corticobasal degeneration, and Pick's disease, Tau ligand-1 shows high affinity for aggregated tau protein, with equilibrium dissociation constants (KD) ranging from 1 to 3.8 nM. It holds potential as a positron emission tomography (PET) tracer for imaging studies of tau-related diseases within the central nervous system.

5β-Cholestane-3α,7α,12α,25-tetrol is a type of bile acid alcohol that activates the G protein-coupled bile acid receptor 1 (GPBAR1), also known as TGR5, in HEK293 cells that express the human receptor, at concentrations between 0.1 to 10 µM. In individuals with cerebrotendinous xanthomatosis (CTX), a progressive metabolic leukodystrophy, levels of this compound are elevated in the blood, feces, and bile.

Brr2-IN-3 (Brr2 Inhibitor C9) is an allosteric inhibitor of the spliceosomal RNA helicase Brr2. Brr2 is implicated in autosomal-dominant retinitis pigmentosa (a group of progressive retinal degenerative disorders).

Ganglioside GD3 is synthesized by the addition of two sialic acid residues to lactosylceramide and can serve as a precursor to the formation of more complex gangliosides by the action of glycosyl- and sialyltransferases. It induces apoptosis in HuT-78 cutaneous T cell lymphoma cells in a concentration-dependent manner and disrupts the mitochondrial membrane potential when used at a concentration of 200 μM. Expression of ganglioside GD3 in GD3-negative SK-MEL-28-N1 malignant melanoma cells increases both cell proliferation and invasion in vitro. Ganglioside GD3-deficient adult mice exhibit progressive loss of the neural stem cell (NSC) pool and impaired neurogenesis. Ganglioside GD3 mixture contains ganglioside GD3 molecular species with C18:1 and C20:1 sphingoid backbones.

Ezeprogind disulfate is a neurotrophic inducer. Ezeprogind disulfate targets all causes of neurodegeneration including Abeta protein or tau protein. Ezeprogind disulfate can be used for the research of neurological disorders, including progressive supranu

3'-sulfo Galactosylsphingosine is a form of sulfatide that is lacking the fatty acyl group. It decreases migration and adhesion of B35 neuroblastoma cells and increases cell rounding when used at a concentration of 20 μM. It also inhibits PKC and cytochrome c oxidase activity when used at concentrations of 150 and 50-100 μM, respectively. 3'-sulfo Galactosylsphingosine accumulates in patients with metachromatic leukodystrophy, a lysosomal storage disorder characterized by arylsulfatase A (ASA) deficiency leading to progressive demyelination and neuromotor deficits. In mice lacking ASA, levels of 3'-sulfo galactosylsphingosine increase after one month of age followed by demyelination and neuromotor deficits. 3'-sulfo Galactosylsphingosine has been used as a standard for the quantification of 3'-sulfo galactosylsphingosine by LC-MS.

6-Aminophenanthridine is an antiprion agent. It inhibits prion formation in yeast- and mammalian-based screening assays when used alone and, to a greater extent, when used in combination with the α2-adrenergic receptor agonist guanabenz . 6-Aminophenanthridine (300 μM) inhibits protein folding activity of the ribosome (PFAR) by directly competing with protein substrates for the active site and decreases the yield of refolded protein without affecting the refolding rate. It prevents progressive wing position defects in a Drosophila model of oculopharyngeal muscular dystrophy (OPMD) when larvae are raised on medium containing doses ranging from 300 to 400 μM and in adults following dietary administration of 1-3 mM doses. 6-Aminophenanthridine also reduces muscle degeneration and decreases the number of nuclear inclusions in thoracic muscle in a Drosophila model of OPMD.

Ezeprogind (AZP2006), a lysotropic molecule associated with PGRN production, binds to PSAP (the cofactor of PGRN) and inhibits TLR9 receptors, which are normally responsible for proinflammation when activated. Ezeprogind has been used in Alzheimer's disease research.

Laquinimod (ABR-215062) sodium is a carboxamide derivative administered orally that serves as a powerful immunomodulator, designed to prevent inflammation and neurodegeneration within the central nervous system. This compound effectively diminishes the activation of astrocytic NF-κB, offering protection against Cuprizone-induced demyelination. It shows promise for the treatment of both relapsing-remitting (RR) and chronic progressive (CP) forms of multiple sclerosis (MS; RRMS or CPMS), in addition to its potential applications in the study of neurodegenerative diseases [1] [2] [3] [4].

LDN-193189 Tetrahydrochloride (DM-3189 Tetrahydrochloride) is a selective inhibitor of BMP type I receptors, inhibiting ALK2 and ALK3 (IC₅₀ = 5/30 nM), with weaker activity against ALK4, ALK5, and ALK7, and can be used for research into progressive osseous fibrodysplasia.

Laquinimod (LAQ) is an effective immunomodulator, which is currently under development in phase III trials for the treatment of multiple sclerosis as an oral therapy.

Aurora kinase-IN-1, a potent aurora kinase inhibitor, modulates cell cycle progression and induces apoptosis by upregulating G1 cell cycle inhibitory proteins (including p21 and p27), elevating G1 progressive cyclin D1, and downregulating G1-to-S progressive cyclins, thereby arresting the cell cycle at the G1 S boundary. It stands as a promising lead compound for the development of chemotherapeutic agents.

1-Linoleoyl lysophosphatidic acid (1-linoleoyl LPA), a glycerophospholipid with linoleic acid at its sn-1 position, acts as an LPA2 receptor agonist and is the predominant LPA form in both mouse and human plasma. This compound preferentially boosts intracellular calcium in Sf9 cells expressing the LPA2 receptor (EC50 = ~10 nM), compared to those with LPA1 and LPA3 receptors (EC50s = ~200 and ~80 nM, respectively). Notably, 1-linoleoyl LPA serum concentrations are elevated in mice with NCTC clone 2472 tumors, while its plasma levels are reduced in primary progressive multiple sclerosis patients—unlike in those with relapsing-remitting multiple sclerosis—and inversely associate with the severity of neurological deficits.

Zilurgisertib fumarate (INB-000928 fumarate, NBU-928 fumarate), a selective ALK-2 inhibitor, is in development for the treatment of ectopic ossification and progressive fibrodysplasia ossificans.

N-Ac-4-S-CAP is a compound that exhibits selective cytotoxicity towards melanin-producing cells in mouse hair follicles, causing 98% depigmentation. Within four hours of intraperitoneal injection, visible changes occur in these melanocytes, including the aggregation of melanin granules and nuclear condensation. Electron microscopy reveals progressive destruction of these cells, evidenced by swelling of membranous organelles, nuclear condensation, and cytoplasmic vacuolization, leading ultimately to complete cell death. N-Ac-4-S-CAP selectively targets active melanin-producing melanocytes but does not appear to affect precursor or dormant melanocytes. These properties suggest potential applications for N-Ac-4-S-CAP in melanoma suppression or skin whitening.

RS-25344 is a potent and selective phosphodiesterase (PDE) 4 inhibitor. It inhibits eosinophil chemotaxis and increases progressive motility of spermatozoa in vitro.

LSTc (LS-tetrasaccharide c), a human lactooligosaccharide found on glycoproteins and glycolipids, serves as a specific recognition motif for the human JC polyomavirus (JCV). This compound holds considerable potential for research into progressive multifocal leukoencephalopathy (PML) [1].