Efficacy

Calcort (Deflazacort) Efficacy in Duchenne Muscular Dystrophy (DMD)

Calcort, known generically as deflazacort, is a corticosteroid medication that has been studied for use in Duchenne Muscular Dystrophy (DMD). DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. Corticosteroids like deflazacort are considered a mainstay in the management of DMD due to their ability to slow the progression of muscle weakness. Clinical trials have demonstrated that deflazacort can improve muscle strength and function in individuals with DMD. It is thought to exert its beneficial effects by reducing inflammation, decreasing muscle protein breakdown, and improving the repair of muscle cells.

The efficacy of deflazacort in DMD was highlighted in a study that showed patients on the drug had a slower decline in muscle strength compared to those not receiving the treatment. Additionally, deflazacort has been associated with a delay in the loss of ambulation, meaning that patients could walk independently for a longer period. Despite these benefits, it is important to note that deflazacort is not a cure for DMD and does not halt disease progression entirely. Instead, it is used to manage symptoms and improve quality of life.

Amondys 45 (Casimersen) Efficacy in Duchenne Muscular Dystrophy (DMD)

Amondys 45, with the active ingredient casimersen, is an antisense oligonucleotide approved for the treatment of DMD in patients who have a confirmed mutation of the dystrophin gene amenable to exon 45 skipping. This medication works by binding to exon 45 of the dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing. By skipping exon 45, Amondys 45 allows for the production of an internally truncated, yet functional, dystrophin protein, which is missing in patients with DMD.

The approval of Amondys 45 was based on the increase in dystrophin production in skeletal muscle observed in patients treated with the drug. The presence of dystrophin is critical for muscle fiber integrity, and the increase in dystrophin production is presumed to provide a clinical benefit. In clinical studies, patients treated with Amondys 45 demonstrated a statistically significant increase in dystrophin levels compared to baseline, which suggests a potential for slowing disease progression. However, it is essential to acknowledge that the clinical benefit of Amondys 45, such as improvement in motor function or delay in disease progression, has not been definitively proven, and further studies are ongoing to determine its long-term efficacy.