|
T7837 |
SR8278
|
|
Others
|
|
SR8278 is an REV-ERBα antagonist(EC50 = 0.47 µM), blocking activation of the receptor by the synthetic agonist GSK 4112 |
|
T39516 |
Bocidelpar
|
|
PPAR
|
|
Bocidelpar is a modulator of PPARδ with an EC50 of 7.80 nM and improves mitochondrial biogenesis and function in Duchenne Muscular Dystrophy muscle cells. |
|
T17217 |
Vamorolone
|
VBP15 |
Glucocorticoid Receptor
,
NF-κB
|
|
Vamorolone (VBP15) is an orally active dissociative steroidal anti-inflammatory drug and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone displays effective NF-κB inhibition an... |
|
T16545 |
Pivanex
|
Pivalyloxymethyl butyrate,AN-9 |
Apoptosis
,
HDAC
,
Bcr-Abl
|
|
Pivanex (Pivalyloxymethyl butyrate) is an orally active HDAC inhibitor and an antimetastatic and antiangiogenic agent. Pivanex downregulates the Bcr-Abl protein and enhances apoptosis. |
|
T4641 |
Branaplam
|
LMI 070,NVS-SM1 |
Others
,
Potassium Channel
,
DNA/RNA Synthesis
|
|
Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator. |
|
T3424 |
Ezutromid
|
BMN 195,VOX-C1100,SMT C1100 |
Others
|
|
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 ... |
|
T13491 |
Neuromuscular-targeting compound 1
|
|
Others
|
|
Neuromuscular-targeting compound 1 can be used to study muscular dystrophy and neuromuscular-related diseases. |
|
T10059 |
Neuromuscular Disorder-Targeting Compound 1
|
|
Others
|
|
Neuromuscular Disorder-Targeting Compound 1 is a compound used in the research of neuromuscular disorders, for example, symptoms of chronic fatigue syndrome and fibromyalgia syndrome. |
|
T7722 |
Feprazone
|
Prenazone,DA 2370,DA2370,DA-2370 |
Others
|
|
Feprazone (DA-2370) possesses anti-inflammatory and antiadipogenic properties. Feprazone can be used in studies about the treatment of joint and muscular pain. |
|
T3336 |
Rg3039
|
PF-06687859 |
Others
|
|
Rg3039 (PF-06687859) is a potent DcpS inhibitor. DcpS is a therapeutic target for spinal muscular atrophy. Spinal muscular atrophy (SMA) is caused by deletion or mutation of both copies of the SMN1 gene which produces an... |
|
T12718L |
RG7800
|
RO6885247 |
Others
,
DNA/RNA Synthesis
|
|
RG7800 (RO6885247) has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier. |
|
T12935 |
SMN-C3
|
MV8T2MCK57 |
DNA/RNA Synthesis
|
|
SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA). |
|
T2553 |
Etofenamate
|
|
COX
|
|
Etofenamate is a non-steroidal anti-inflammatory drug (NSAID) used for the treatment of joint and muscular pain. |
|
T1805 |
Ataluren
|
PTC124 |
CFTR
,
Autophagy
|
|
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 olde... |
|
T9821 |
ART-CHEM-BB B025267
|
|
Others
|
|
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. |
|
T0319 |
Suxibuzone
|
Calibene,Suxibuzonum,Suxibuzona |
COX
|
|
Suxibuzone (Suxibuzona) is a drug used for joint and muscular pain. It is a prodrug of the non steroidal anti inflammatory drug phenylbutazone, and is commonly used in horses. |
|
T77445 |
Apitegromab
|
SRK-015 |
Opioid Receptor
|
|
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. |
|
T72072 |
2-benzylsulfanyl-6-methoxy-4-methylquinazoline
|
|
ROS
|
|
2-benzylsulfanyl-6-methoxy-4-methylquinazoline can be used as a TRPML modulator for the treatment of diseases associated with TRPML activity, such as lysosomal storage disease, muscular dystrophy, oxidative stress or rea... |
|
T15945 |
MA-0204
|
|
PPAR
|
|
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 treatmen... |
|
T0511 |
Tolperisone hydrochloride
|
Muscalm,Midocalm,Tolperisone HCl |
Sodium Channel
|
|
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 encephalomyel... |
|
T72073 |
AKOS037652256
|
|
|
|
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, oxi... |
|
T73475 |
SMN-C2
|
|
DNA/RNA Synthesis
|
|
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... |
|
T21792 |
CGP 3466B maleate
|
Omigapil (Maleate),Omigapil maleate |
Apoptosis
|
|
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 male... |
|
T22232 |
Alfuzosin
|
|
|
|
Alfuzosin, a quinazoline derivative, is a selective and competitive antagonist of the α1 adrenergic receptor, primarily used to manage benign prostatic hyperplasia (BPH). It effectively targets and mitigates alpha1-adren... |
|
T31168 |
D153249
|
D 153249,D-153249 |
|
|
D153249 is a promoter of spinal muscular atrophy (SMA). |
|
T38554 |
Eteplirsen
|
AVI 4658 |
|
|
Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research for Duchenne muscular dystrophy. |
|
T39517 |
Suvodirsen
|
WVE-210201 |
|
|
Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD). |
|
T7710L |
Mofebutazone sodium
|
BRN0213056,BRN 0213056,BRN-0213056 |
|
|
Mofebutazone sodium is, a nonsteroidal antiinflammatory drug (NSAID), is used to treat joint and muscular pain. |
|
T34664 |
SMN2-Stablizer-27
|
SMN2-Stablizer 27,SMN2 Stablizer-27 |
|
|
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). |
|
T83176 |
Activated C Subunit
|
|
|
|
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 ... |
|
T83173 |
Activated T Subunit
|
|
|
|
Activated T Subunit is employed in synthesizing exon-jumping oligomer conjugates that target specific sites within the human anti-muscular atrophy protein gene to induce exon 51 skipping, a potential avenue for muscular ... |
|
T83175 |
Activated DPG Subunit
|
|
|
|
Activated DPG subunit facilitates the synthesis of exon jumping oligomer conjugates targeting specific sites within the human anti-muscular atrophy protein gene to induce exon 51 skipping, a potential research avenue for... |
|
T38860 |
Golodirsen
|
Golodirsen,SRP-4053 |
|
|
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD). |
|
T81693 |
NCP2 Anchor
|
|
|
|
NCP2 Anchor is employed in synthesizing exon jumping oligomer conjugates that align with specific target sequences within the human anti-muscular atrophy protein gene to promote exon 52 skipping, facilitating research in... |
|
T39455 |
Viltolarsen
|
NS-065,NCNP-01 |
|
|
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically targets the splicing of exon 53 in the dystrophin gene. It is utilized in research related to Duchenne muscular ... |
|
T83177 |
Activated A Subunit
|
|
|
|
Activated A Subunit is instrumental in synthesizing exon skipping oligomer conjugates, which are designed to specifically pair with chosen sequences within the human anti-muscular atrophy protein gene, thereby promoting ... |
|
T34426 |
RTC14
|
Read-through compound-14,RTC 14,RTC-14,Read-through compound 14 |
|
|
RTC14, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy. |
|
T34425 |
RTC13
|
Read-through compound 13,RTC 13,RTC-13 |
|
|
RTC13, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy. |
|
T66481 |
TG-693
|
|
|
|
TG693, an orally available inhibitor of CLK1, promotes the skipping of the endogenous mutated exon 31 in Duchenne muscular dystrophy (DMD) patient-derived cells and increased the production of the functional exon 31-skip... |
|
T83174 |
Activated EG3 Tail
|
|
|
|
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 ... |
|
T61645 |
Utrophin modulator 1
|
|
|
|
Utrophin Modulator 1 (UM1) is a highly effective agent that upregulates utrophin protein levels, displaying an EC 50 of 0.11 μM. Its application in the investigation of Duchenne Muscular Dystrophy (DMD) has been well-est... |
|
T79918 |
α7β1 integrin modulator-1
|
|
Integrin
|
|
α7β1 Integrin Modulator-1 is a potent modulator with research potential for muscular dystrophy [1]. |
|
T14129 |
Adenylosuccinic acid
|
Aspartyl adenylate,Adenylosuccinate |
Others
|
|
Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid has the potential for the study of duchenne muscu... |
|
T78046 |
RG7800 tetrahydrochloride
|
RO6885247 tetrahydrochloride |
DNA/RNA Synthesis
|
|
RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal m... |
|
T33595 |
Naproxcinod
|
AZD3582,Nitronaproxen,AZD-3582,Naproxen-N-butyl nitrate,HCT3012,AZD 3582 |
|
|
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... |
|
T73321 |
CLK1-IN-2
|
|
|
|
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 HI... |
|
T60261 |
Clorprenaline
|
|
|
|
Clorprenaline, a potent β2-adrenergic agonist, effectively promotes muscular mass growth in animals while reducing fat accumulation, making it a promising lean meat-enhancing feed additive [1]. |
|
T38859 |
Casimersen
|
SRP-4045 |
|
|
Casimersen (SRP-4045) is an antisense oligonucleotide, specifically belonging to the phosphorodiamidate morpholino oligomer subclass. It binds to exon 45 of dystrophin pre-mRNA, effectively restoring the open-reading fra... |
|
T82778 |
Cardioexcitatory peptide 1
|
|
|
|
Cardioexcitatory peptide 1, a potent neuropeptide derived from Achatina atria, exerts significant excitatory effects on cardiac function, additionally influencing the motility of muscular tissues and neural activities [1... |
|
T60594 |
OX01914
|
|
|
|
OX01914 is a water-solube and permeable modulator of utrophin that can upregulate utrophin protein levels with an EC 50 of 20.5 μM. OX01914 can be used in Duchenne muscular dystrophy (DMD) study [1]. |
