Golodirsen research and development background and analysis of overseas drug purchase channels

Golodirsen (golodirsen) is an exon skipping therapy drug for Duchenne muscular dystrophy (DMD), developed by Sarepta Therapeutics. Its main mechanism of action is to restore part of the function of the muscular dystrophy gene by skipping exon 53, thereby promoting the expression of some functional muscle proteins. This mechanism provides targeted treatment options for DMD patients, especially for patients with DMD gene exon 53 deletion.

In terms of research and development background, Gorodison's research and development began with the exploration of combining gene therapy and oligonucleotide therapy, and its safety and effectiveness have been verified through years of clinical trials. Clinical data shows that the drug can increase the expression of some muscle proteins during long-term treatment, delay the decline of muscle function, and have a certain protective effect on patients' exercise ability and quality of daily life. During the research and development process, focus was placed on dose optimization, long-term tolerance and genotype matching to ensure both efficacy and safety.

Regarding overseas drug purchase channels, Golodisin has not yet been launched in China, so domestic patients can only obtain it through formal overseas channels. Overseas original drugs are mainly provided through Sarepta Therapeutics or its authorized imported medical institutions. The price is relatively high, with a box of drugs costing more than a thousand US dollars. During the purchase process, international prescription drug management regulations must be followed, including providing legal prescriptions, medical certificates, and cross-border drug transportation compliance procedures to ensure that the sources of drugs are regular and safe to use.

In clinical practice, patients and their families should fully understand the indications, dosage regimen and long-term management requirements of the drug. Since Golodisen is a gene-targeted therapy, professional doctors must be used to regularly assess muscle function, gene expression and potential adverse reactions during use. In addition, combined with rehabilitation training, nutritional management and life intervention, the clinical benefits of drugs can be maximized, delaying the progression of DMD patients and improving their quality of life.

Keyword tags: Gorodison, Duchenne muscular dystrophy, exon skipping therapy, overseas drug purchase, gene therapy

References:https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-approval-vyondys-53tm