muscular

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

Neuromuscular-targeting compound 1 can be used to study muscular dystrophy and neuromuscular-related diseases.

Neuromuscular Disorder-Targeting Compound 1 is utilized in research for neuromuscular disorders, including chronic fatigue syndrome and fibromyalgia syndrome symptoms.

CGP 3466B maleate (Omigapil maleate) is an orally bioavailable GAPDH nitrosylation inhibitor. Omigapil maleate abrogates Aβ1-42-induced tau acetylation, memory impairment, and locomotor dysfunction in mice. Omigapil maleate has the potential for the research of Alzheimer's disease. Omigapil maleate (CGP3446B maleate) is a apoptosis inhibitor. Omigapil maleate can be used for the research of congenital muscular dystrophy (CMD).

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

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).

Edasalonexent (CAT-1004) is an orally available NF-κB inhibitor designed to improve Duchenne muscular dystrophy.

Methyl salicylate (Gaultheria oil) is an organic ester naturally produced by many species of plants, particularly wintergreens. For acute joint and muscular pain, methyl salicylate is used as a rubefacient and analgesic in deep heating liniments.

Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.

Tolperisone hydrochloride (Muscalm), a centrally acting muscle relaxant, is used in the therapy of pathologically increased tone of the cross-striated muscle resulted by neurological diseases (damage of the encephalomyelitis, myelopathy, multiple sclerosis, pyramidal tract) and of spastic paralysis and other encephalopathies manifested with muscular dystonia.

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

SR8278 is an REV-ERBα antagonist(EC50 = 0.47 µM), blocking activation of the receptor by the synthetic agonist GSK 4112

Etofenamate is a non-steroidal anti-inflammatory drug (NSAID) used for the treatment of joint and muscular pain.

ART-CHEM-BB B025267 is the upregulator of utrophin production with EC50 of 1.8 μM and can be used in research on the treatment of Duchenne muscular dystrophy.

MA-0204 is a highly selective and orally available peroxisome proliferator-activated receptor δ (PPARδ) modulator (EC50s: 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively). It is a potential treatment for Duchene Muscular Dystrophy (DMD).

Ezutromid (BMN 195) is a translation modulator of Small utrophin (EC50: 0.4 uM). Ezutromid is an orally bioavailable small molecule that transcriptionally upregulates the utrophin gene, increasing both utrophin mRNA and protein. Through this action, Ezutromid increases muscle function in dystrophin-deficient MDX mice, a mouse model of muscular dystrophy.

AKOS037652256 can be used as a TRPML modulator for the treatment of diseases associated with TRPML activity such as lysosomal accumulation disorders, muscular dystrophy, common age-related neurodegenerative diseases, oxidative stress or reactive oxygen species (ROS)-related diseases and ageing.

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

RTC13 is a read-through reagent that restores anti-myasthenia gravis protein expression in the mdx mouse model of Duchenne muscular dystrophy (DMD).

Naproxcinod, a nitric oxide-releasing NSAID derivative, significantly improved skeletal muscle strength and fatigue resistance in sedentary and exercise mice, reducing inflammatory infiltration and fibrotic deposition in the myocardium and diaphragm. In a

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.

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].

H-D-MeAla-EtVal-Val-MeLeu-Ala-D-Ala-MeLeu-MeLeu-MeVal-MeBmt(OAc)-Abu-O-CH2-CH2-NHMe is a derivative of cyclosporin A, lacking immunosuppressive properties, with potential applications in congenital muscular dystrophy research [1].

CLK1-IN-2, a metabolically stable Clk1 inhibitor, exhibits selective activity for Clk1 with an IC50 value of 1.7 nM. It is applicable in researching tumor, Duchenne's muscular dystrophy, and viral infections including HIV-1 and influenza.