Golodirsen drug introduction and detailed analysis of indications, mechanism of action and clinical application

Golodirsen is a new type of antisense oligonucleotide drug, mainly used to treat Duchenne Muscular Dystrophy (DMD) caused by the deletion of exon 53 of the DMD gene. DMD is a progressive muscle degenerative disease caused by chromosome-linked recessive inheritance of The emergence of Golodisen provides a targeted treatment option for DMD patients, especially those with exon deletion of 53. Its clinical application is highly individualized.

Glodisen’s mechanism of action belongs to antisense oligonucleotide therapy (antisense oligonucleotide, ASO), by selectively binding to the target DMD gene’s mRNA, causing cells to transcribe In the process, the missing 53 exon is skipped, thereby generating dystrophin (dystrophin) whose function is partially restored. Although the dystrophin produced is still incomplete, it is sufficient to improve muscle fiber structure and function, thus slowing the progression of the disease. Compared with traditional treatments, Golodisen has the advantages of strong targeting and relatively low side effects. However, the efficacy usually requires long-term use to appear, and the efficacy is affected by individual differences and genotypes of patients.

In clinical studies, Golodisen was mainly administered via intravenous injection, with the recommended dose being 30 mg/kg once a week. Studies have shown that long-term use of Golodisen can increase the expression level of dystrophin in the muscles of some DMD patients and improve the maintenance of muscle strength and motor function. In clinical observations, Golodisen has generally good safety in terms of blood indicators, liver, kidney and heart functions, but some side effects may still occur, such as injection site reactions, mild increases in urinary protein and changes in blood pressure. Physicians need to regularly monitor the patient's liver and kidney function and muscle condition during use to ensure safety and efficacy.

The clinical application of Golodisen also requires individualized evaluation based on the patient's age, disease course and genotype. Early diagnosis and early use are often recommended to slow disease progression and improve quality of life. For families and medical staff, the application of Golodisen involves not only the drug itself, but also comprehensive measures such as long-term rehabilitation training, respiratory support and cardiac management to achieve an overall therapeutic effect. Although Golodisen is not currently on the market in China, it has been approved in overseas markets. Patients can obtain the drug through formal channels and must strictly follow the guidance of professional doctors for long-term medication and follow-up monitoring. In summary, Golodisin represents the forefront of gene-targeted therapy for DMD and provides a feasible intervention for patients with specific genotypes, but comprehensive clinical management is still required to obtain the best therapeutic effect.

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