Givinostat hydrochloride 

Store conditions: -20°C

Solubility in DMSO: 45.8 (Maximum concentration mg/mL)

Solubility in DMSO: 100.01 (Maximum concentration mM)

Form: Solid

Color: White to Off-white

Cell Experiment

Solubility in DMSO: 175 mg/mL (382.14 mM; ultrasonication required for dissolution; hygroscopic DMSO significantly affects the solubility of the product, please use freshly opened DMSO)

Preparing Stock Solutions：

*Select an appropriate solvent for the preparation of stock solution based on the solubility of the product in different solvents. Once prepared, aliquot and store the solution to avoid product failure caused by repeated freeze-thaw cycles.

Storage conditions and shelf life of the stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, protected from moisture). When stored at -80°C, use within 6 months; when stored at -20°C, use within 1 month.

The efficacy givinostat was demonstrated in a randomized, double-blind, placebo-controlled trial wherein muscle function was evaluated by measuring the change from baseline to 18 months in the time taken to ascend four stairs. Patients treated with givinostat showed statistically significant less decline in the time it took to climb four stairs compared to placebo - the mean change was 1.25 seconds for patients receiving givinostat compared to 3.03 seconds for patients receiving placebo.

Givinostat causes QTc interval prolongation and should be used with caution in patients with underlying cardiac disease or in patients who are taking concomitant medications that may prolong the QT interval.

Givinostat is a histone deacetylase inhibitor. The precise mechanism by which givinostat exerts its therapeutic effects in patients with DMD is unknown. Histone deacetylases (HDACs), as the name implies, regulate the deacetylation of various proteins. The acetylation and deacetylation of histone proteins causes an increase or decrease in gene expression, respectively, with the latter function governed by HDACs. The balance between levels of histone acetylation and deacetylation plays a key role in the modulation of gene transcription and governs numerous developmental processes, being involved in the regulation of various genes associated with signal transduction, cell growth, and cell death, as well as diseases like cancers.1 HDACs can deacetylate non-histone proteins, such as p53, thereby also regulating their activity.