How much does a box of golodirsen cost, as well as market reference prices and drug purchasing channels

Golodirsen (Golodirsen) is an exon 53 skipping therapy drug for Duchenne Muscular Dystrophy (DMD), for patients with specific gene mutations. This drug is not currently available in mainland China, so domestic patients cannot obtain prescriptions through formal channels. Golodirsen The therapeutic goal is to delay the loss of muscle function by changing gene expression so that the body can produce partially functional dystrophin. Clinical data show that for pediatric patients who meet the indications, long-term use can slow down disease progression to a certain extent, but the efficacy still needs to be evaluated based on individual circumstances.

Regarding price and market reference information, Golodirsen costs more when listed overseas. According to public information, the annual cost of treatment options in the United States can be as high as approximately $748,000 based on recommended doses. On some unofficial channels or online channels, there are also quotations of "about more than a thousand dollars per box", but such channels lack quality and safety guarantees, and the prices fluctuate greatly, and they do not represent regular market pricing. Therefore, if you are considering purchasing overseas, you need to fully understand the legality and source of the drug and avoid illegal or riskier sources.

Golodirsen ’s drug purchase channels are mainly concentrated in overseas countries where it has been approved for marketing, such as the United States. Patients usually need hospital infusions and professional medical institutions to administer drugs, and insurance or medical payment systems can bear part of the cost. If domestic patients want to use this drug, they need to pay attention to international clinical trials or obtain it through legal international medical cooperation projects, and at the same time follow strict medical management and prescription regulations to ensure the safety and effectiveness of the drug.

Overall, Golodirsen ’s high price and complicated drug purchase channels have restricted its domestic use. When patients and family members pay attention to drugs, they should focus on understanding their indications, therapeutic effects and safety, and formulate a reasonable treatment plan based on doctor's recommendations. Although it is currently not directly available in China, with future drug approval and marketing progress, it is expected to bring accessible treatment options to more patients.

Reference materials:https://en.wikipedia.org/wiki/Golodirsen