Duchenne muscular dystrophy (DMD), a rare genetic disorder impacting 1 in 5,000 male births globally, poses significant challenges. Viltepso, classified as an antisense oligonucleotide, is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a verified mutation of the DMD gene amenable to exon 53 skipping. The approval for this indication is granted under accelerated approval, substantiated by the observed increase in dystrophin production within skeletal muscle among patients treated with Viltepso.
DMD Insights and Conventional Approaches
DMD, a progressive muscle-wasting ailment rooted in a DMD gene mutation resulting in dystrophin deficiency, prompts exploration beyond traditional treatments like corticosteroids. While these drugs can moderately slow muscle weakening and enhance strength, their limitations underscore the urgency for more potent therapies.
Viltepso’s Mechanism
Viltepso, an antisense oligonucleotide, directs exon skipping in the DMD gene, facilitating the production of a truncated dystrophin form. This exon-skipping strategy aims to elevate dystrophin levels in skeletal muscles, mitigating the progression of muscle weakness in DMD patients.
Dosage And Administration
Prior to the initiation of Viltepso, a comprehensive evaluation encompassing serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio is imperative. The prescribed dosage stands at 80 milligrams per kilogram of body weight, to be administered intravenously over a duration of 60 minutes. In instances where the requisite volume of Viltepso is below 100 mL, it necessitates dilution in 0.9% Sodium Chloride Injection, USP. The formulation is available as an injectable solution in a 250 mg/5 mL (50 mg/mL) single-dose vial.
Clinical Advantages of Viltepso
Clinical trials affirm Viltepso’s capacity to heighten dystrophin production in skeletal muscles, manifesting as noteworthy enhancements in timed function tests. While not a curative solution, Viltepso exhibits potential in slowing disease progression, potentially prolonging independent mobility, eating, and breathing for individuals with DMD. Common clinical trial side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia.
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Individuals seeking to import medication are required to provide a valid prescription, proof of identity, information about their healthcare professional, and current address. The Sansfro team initiates the import license application, ensuring access to the necessary medication upon government approval. To inquire about Viltepso price in India and understand the importation procedure, interested individuals can contact the Patient Support Team at (+91) 9315705373 or via email at help@sansfro.com.
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