Explanation of domestic drug purchase channels and formal acquisition methods of golodirsen

Golodirsen (golodirsen) is an exon skipping therapy drug for patients with Duchenne muscular dystrophy (DMD). It is mainly targeted at patients who meet the indications in the DMD gene. The drug can regulate mRNA splicing, causing abnormal DMD gene expression to produce partially functional dystrophin, thereby delaying disease progression. Due to its special therapeutic effect and pertinence, it has not yet been approved for marketing in China, so domestic patients need to obtain it through special channels.

in the country, Golodisen cannot be purchased directly through hospitals or pharmacies. If patients want to use it, they need to purchase the original medicine through formal overseas channels. Common channels include overseas authorized pharmacies or cross-border medical service platforms, but it is necessary to ensure that the channels are legal and the sources of drugs are reliable to avoid the risk of counterfeit or unverified drugs. Before purchasing, the indications should be evaluated by a professional doctor and the relevant prescription and medical certificate should be provided.

The price of overseas Golodisin is relatively high, about more than one thousand US dollars per box. The specific price is affected by the supplying country and exchange rate changes. Since the drug is an injection or intravenous preparation, patients still need to use it under the guidance of a medical institution after obtaining the drug to ensure the correct administration method, accurate dosage, and regular monitoring of blood, liver and kidney function and other indicators.

Formal ways to obtain Golodisin also include participating in clinical trials or special import approval projects. In some areas of the country, patients can apply to the drug administration department for permission to use imported drugs through hospitals, but they need to provide detailed medical records and proof of indications. The entire process must strictly follow regulatory requirements to ensure the safety and legality of medication.

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

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