Catalysts are substances that increase the reaction rate of a chemical reaction without being consumed in the process. 88-17-5, Name is 2-(Trifluoromethyl)aniline, SMILES is NC1=CC=CC=C1C(F)(F)F, belongs to pyridazines compound. In a document, author is Cheung, Atwood K., introduce the new discover, Name: 2-(Trifluoromethyl)aniline.
Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA), a rare neuromuscular disorder, is the leading genetic cause of death in infants and toddlers. SMA is caused by the deletion or a loss of function mutation of the survival motor neuron 1 (SMN1) gene. In humans, a second closely related gene SMN2 exists; however it codes for a less stable SMN protein. In recent years, significant progress has been made toward disease modifying treatments for SMA by modulating SMN2 pre-mRNA splicing. Herein, we describe the discovery of LMI070/branaplam, a small molecule that stabilizes the interaction between the spliceosome and SMN2 pre-mRNA. Branaplam (1) originated from a high-throughput phenotypic screening hit, pyridazine 2, and evolved via multiparameter lead optimization. In a severe mouse SMA model, branaplam treatment increased full-length SMN RNA and protein levels, and extended survival. Currently, branaplam is in clinical studies for SMA.
The proportionality constant is the rate constant for the particular unimolecular reaction. the reaction rate is directly proportional to the concentration of the reactant. I hope my blog about 88-17-5 is helpful to your research. Name: 2-(Trifluoromethyl)aniline.
Reference:
Pyridazine – Wikipedia,
,Pyridazine | C4H4N2 – PubChem