atrophy

Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

Epristeride (ONO-9302) is an orally active, selective and non-competitive steroidal 5-α-reductase isoform 2 inhibitor that inhibits SR isoform 2.Episteride reduces prostate size and improves symptoms in men with BPH.Episteride induces atrophy and apoptosis of the ventral prostate in rats.

SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).

OT-551 HCl is an NF-Κb inhibitor, a disubstituted hydroxylamine with antioxidant properties can be used for the treatment of cataracts and age-related macular degeneration and geographic atrophy (GA).

SMN-C2 is a selective regulator of SMN2 gene splicing, a risdiplam analogue, a selective RNA-binding ligand that regulates pre-mRNA splicing and acts by binding to SMN2 pre-mRNA.SMN-C2 has the potential to be used in the study of spinal muscular atrophy (SMA).

Fispemifene (HM-101) is an estrogen receptor modulator (SERM) used to treat genitourinary syndromes and vaginal atrophy.

Sabinene is a natural product that has antifungal and anti-inflammatory properties

Pivanex (Pivalyloxymethyl butyrate) is an orally active HDAC inhibitor and an antimetastatic and antiangiogenic agent. Pivanex downregulates the Bcr-Abl protein and enhances apoptosis.

Promestriene (3-propyl ethyl) (INN) is a synthetic estrogen and glucocorticoid used topically in a 1% cream formulation. It is described as a tropic agent and antiseborrheic.

Ospemifene (FC-1271a) is a new selective non-hormonal estrogen receptor modulator (SERM) that is used for the treatment of moderate to severe dyspareunia, a symptom of vulvar and vaginal atrophy, due to menopause. FDA approved on February 26, 2013.

D153249 is a promoter of spinal muscular atrophy (SMA).

Myostatin Inhibitory Peptide 2 (compd 2) TFA is a potent inhibitor of muscle growth suppressant with a dissociation constant (Kd) of 35.9 nM. It is utilized in the study of muscle atrophy diseases.

Biotinoyl tripeptide-1 can produce hair follicles by promoting scalp micro-circulation and reduce follicle atrophy and aging.

The Survival of Motor Neurons (SMN) protein participates in RNA splicing. Decreases in SMN, typically a consequence of defects in the smn1 gene, result in the death of motor neurons and lead to the neurodegenerative disease, spinal muscular atrophy (SMA). Cuspin-1 is a small molecule upregulator of SMN that has been shown in vitro to increase levels of SMN in SMA patient fibroblasts by 50% at 18 μM. Its mechanism of action is thought to involve increased phosphorylation of ERK to initiate Ras-Raf-MEK signaling, which results in an increased rate of SMN translation.

MYLS22 is a first-in-class and selective inhibitor of optic atrophy 1 (OPA1) . MYLS22 can target endothelial OPA1 to curtail tumor growth and inhibits angiogenesis by impinging on NFkB activity and on angiogenic gene expression. It with anti-angiogenesis and anti-cancer activity.

Activated EG3 Tail facilitates the synthesis of exon-skipping oligomer conjugates that target specific sites within the human anti-muscular atrophy protein gene, promoting exon 51 skipping. This compound holds potential for research into muscular dystrophy treatments [1].

Activated C Subunit is utilized in synthesizing exon jumping oligomer conjugates that complement specific target sites within the human anti-muscular atrophy protein gene to promote exon 51 skipping, offering a research avenue for muscular dystrophy [1].

MRS4719, a potent P2X4 receptor antagonist, exhibits a half-maximal inhibitory concentration (IC50) of 0.503 μM for the human P2X4 receptor. Demonstrating both neuroprotective and neuro-rehabilitative effects, it can decrease infarct volume and brain atrophy in an ischemic stroke model. Additionally, MRS4719 mitigates ATP-induced calcium influx in primary human monocyte-derived macrophages, making it a valuable tool for researching ischemic stroke.

CLR01 is a molecular tweezer. CLR01 reduces aggregated, pathologic, and seeding-competent α-synuclein in experimental multiple system atrophy. CLR01 inhibits aberrant superoxide dismutase 1 (SOD1) self-assembly in vitro and in the G93A-SOD1 mouse model of ALS. CLR01 inhibits Ebola and Zika virus infection. CLR01 Stabilizes a Disordered Protein-Protein Interface. CLR01, Modulates Aggregation of the Mutant p53 DNA Binding Domain and Inhibits Its Toxicity.

Apitegromab (SRK-015) is a new myostatin inhibitor, an antibody targeting myostatin precursor, which can be used to study neuromuscular diseases including spinal muscular atrophy.

Pegcetacoplan acetate is a pegylated complement C3 inhibitory peptide that functions by binding to complement component 3 (C3) and its activation fragment C3b. This compound is utilized in the study of complement-mediated diseases, including age-related macular degeneration, C3 glomerulopathy, geographic atrophy (GA), and autoimmune hemolytic anemia.

Biotinoyl tripeptide-1 Acetate can produce hair follicles by promoting scalp micro-circulation and reduce follicle atrophy and aging.

Flumethasone pivalate (Locorten) has antipruritic, anti-inflammatory, and vasoconstrictive properties and can be used in studies about adrenocortical suppression, cutaneous atrophy, plasma transcortin binding, and telangiectasia.

SMN2-Stablizer-27 is a stablizer of survival of motor neuron 2 (SMN2), and a post-translationally stablizing SMN protein for the treatment of spinal muscular atrophy (SMA).