What is golodirsen and analysis of its pharmacological effects

Golodirsen (golodirsen) is a gene-targeted drug used to treat Duchenne Muscular Dystrophy (DMD). It is mainly suitable for patients with exon deletion of DMD gene 53. This drug belongs to the antisense oligonucleotide class (antisense oligonucleotide, ASO () drugs achieve selective splicing of mRNA by targeting specific exons, thereby restoring the expression of some functional dystrophin (dystrophin), providing a targeted treatment option for DMD patients. Golodisin is a precision treatment drug targeting specific mutations of the DMD gene. Compared with traditional symptomatic treatment drugs, it focuses more on intervening in the fundamental mechanism of the disease.

From a pharmacological mechanism of action, Golodisin can induce the cell splicing mechanism to skip the missing or mutated exon by binding to the sequence before and after the 53 exon of the DMD gene, thereby producing mRNA that can express part of functional dystrophin. This mechanism not only delays the degeneration of muscle fibers, but also improves the contractile function of skeletal muscles to a certain extent. Clinical studies have shown that long-term use of Golodisen can increase the expression of dystrophin in skeletal muscle, helping to maintain patients' exercise ability and delay the progression of the disease.

In clinical applications, Golodisen is usually administered via intravenous infusion, and the course of treatment requires long-term maintenance and regular follow-up to evaluate muscle strength and functional indicators. When patients receive drug treatment, they also need to cooperate with rehabilitation training and daily care to achieve the best treatment effect. Clinical trials have shown that the drug has certain advantages in improving motor function and delaying gait loss, and its safety is generally controllable. Common adverse reactions include injection site reactions, mild fever or headache, most of which are mild to moderate and can be alleviated through symptomatic treatment.

In general, Golodisen restores part of dystrophin expression through the exon skipping mechanism, providing precise treatment options for patients with DMDth 53 exon deletion. Its pharmacological effects target the cause of the disease and can delay disease progression and improve motor function, bringing new hope to patients with Duchenne muscular dystrophy. With the continuous deepening of clinical research, the application prospects of Golodisin in personalized gene therapy and long-term management have gradually become clearer, and it has also provided an important reference for the development of gene-targeted drugs.

Reference materials:https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-approval-vyondys-53tm