Analysis of golodirsen side effects, adverse reactions and medication safety recommendations

Golodirsen is a drug used to treat Duchenne Muscular Dystrophy (Duchenne Muscular Dystrophy, DMD) exon 51 skipping antisense oligonucleotide drugs are mainly targeted at DMD patients who carry exon 51 deletion mutations. By regulating messenger RNA splicing, muscle cells can synthesize partially functional dystrophin, thereby delaying muscle degeneration and loss of function. Although Golodisen is a targeted therapy drug, certain side effects and adverse reactions may still occur during clinical use, so patients need to pay special attention to safety issues during medication.

Based on clinical trials and real-world data, the most common adverse reactions of Golodisen include injection site reactions, fever, headache, cough, nasopharyngitis, etc. Injection site reactions manifest as mild redness, swelling, pain or itching, which usually occur within hours to days after injection. Symptoms can generally be relieved by themselves or through cold compress or local care. Headache and fever are usually mild to moderate and can be clinically controlled through symptomatic treatment without affecting long-term medication regimens. Upper respiratory tract symptoms such as cough and nasopharyngitis are common, but are generally not serious, and most patients do not need to stop taking the drug.

Serious adverse reactions that occur less frequently but require attention include abnormal renal function, allergic reactions, and hematological abnormalities. In clinical trials, some patients experienced mild increases in blood creatinine or abnormal urine indicators, suggesting that the kidneys may be mildly affected. Therefore, during long-term medication, doctors usually recommend that patients undergo regular blood routine, renal function and urine routine tests in order to detect abnormalities in time and take intervention measures. For patients who experience obvious allergic reactions or serious adverse events, the drug should be discontinued immediately and treated accordingly.

The drug safety of Golodisen also needs to be combined with the administration method and dosage control. The drug is usually administered via intravenous infusion at a dose prescribed by the doctor every week. The infusion speed and concentration must be strictly controlled to reduce the risk of injection-related adverse reactions. Family members or caregivers should maintain operating standards during the infusion process to ensure the safety of the infusion equipment, drug preparation, and environment. For pediatric patients, parents should pay special attention to observing physical reactions during the infusion process. If difficulty in breathing, rash or abnormal behavior occurs, they should report to the doctor immediately.

In daily life management, patients using Golodisen must also pay attention to a reasonable diet, maintain moderate exercise and adequate rest to assist in the efficacy of the drug. At the same time, simultaneous use with drugs that may damage the kidneys or liver should be avoided to reduce the risk of potential drug interactions. Regular review of muscle function, cardiac function and laboratory indicators following the doctor's guidance is an important step in ensuring the safety and efficacy of long-term medication.

Taken together, Golodisin is a target for exon51 deletionDMDTargeted therapeutic drugs for patients have clear clinical value in delaying disease progression. However, although most of its side effects and adverse reactions are mild to moderate, they still require strict monitoring and standardized management. Through reasonable medication regimens, standardized infusion operations, regular follow-up visits and lifestyle management, patients can ensure drug safety to the greatest extent while optimizing treatment effects. Close cooperation between doctors, patients and their families is the key to ensuring the safety and efficacy of Golodisen during its long-term use.

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