Analysis of golodirsen usage, dosage, treatment duration and clinical guidelines

Golodirsen (Golodirsen) is an antisense oligonucleotide drug targeting Duchenne Muscular Dystrophy (DMD). It is mainly used for DMD patients with specific exon skipping indications. Its mechanism of action is to induce the skipping of the 53 exon of the DMD gene and restore the expression of some functional dystrophin (dystrophin), thereby delaying muscle function degradation and improving disease progression. Golodisen is a gene-level intervention drug that targets patients with specific gene mutations. Therefore, genetic testing is required in clinical applications to confirm whether patients are suitable for use.

In terms of usage and dosage, Golodisen is usually administered by intravenous infusion. The recommended dose is 30 mg/kg once weekly by intravenous infusion. The first dose should be administered in a professional medical facility to monitor for infusion-related adverse reactions, such as fever, rash, or allergic reactions. Subsequent infusions can be continued in qualified hospitals or centers. At the same time, medical staff need to pay attention to changes in the patient's weight in order to dynamically adjust the dose to ensure both efficacy and safety.

Regarding the course of treatment, Golodisen is a long-term maintenance treatment drug. Clinical trials and guidelines recommend continued long-term use to maintain dystrophin expression and delay muscle function decline. Since DMD is a progressive hereditary disease, the efficacy of the drug can only be realized in continuous treatment. Therefore, patients usually require regular weekly infusions for many years. During treatment, muscle function, lung function and heart function need to be regularly evaluated, and hematological indicators and renal function should be monitored at the same time to ensure long-term drug safety.

In terms of clinical guideline analysis, Golodisen has been included in the gene-specific treatment recommendations for DMD and is suitable for patients carrying DMD exon 53 mutations. The guidelines emphasize the importance of individualized treatment and require detailed genetic testing, functional assessment and multidisciplinary team management before treatment. During the treatment process, rehabilitation training, cardiopulmonary function monitoring, and supportive care should be combined to optimize clinical efficacy. In general, Golodisen provides targeted treatment for specific DMD patients, but usage, dosage and long-term monitoring specifications must be strictly followed to achieve maximum clinical benefit.

Keyword tags: Gorodison, Duchenne muscular dystrophy, usage and dosage, course of treatment, clinical guidelines

Reference materials:https://trial.medpath.com/drug/approvals/fda/7256cda011e3383a