Summary

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• Amifampridine (Firdapse) is indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients 6 years of age and older. It has shown significant improvement in muscle strength as measured by the QMG score, compound muscle action potentials amplitude, and in managing LEMS symptoms.
• Four systematic reviews/meta-analyses have been analyzed to gather information on Firdapse's effectiveness against LEMS.
• Amifampridine is generally well tolerated with minimal adverse effects. Common but tolerable side effects include peri-oral and finger paresthesia, along with gastrointestinal reactions.
• Doses below 80 mg/day are recommended to mitigate the risk of serious side reactions, like seizures, indicating a manageable safety profile when dose management guidelines are followed.
• While most studies have primarily addressed adults with LEMS, its indication for use in pediatric patients aged 6 years or older suggests its evaluation across a broader age range.
• Compared to alternative treatments such as earlier forms like 3,4-DAP (not the salt form), amifampridine stands out due to its efficacy over placebo trials, making it an effective first-line treatment option for both adult and pediatric populations suffering from LEMS.