Tag: Evrysdi

Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality, but recent advancements in treatment have transformed the prognosis for patients of all ages. What does SMA medicine do? It works by addressing the underlying genetic cause of the disease, leading to better motor function and improved survival outcomes.

Spinal muscular atrophy (SMA) is a rare genetic disorder affecting approximately 1 in 10,000 live births. The arrival of the pill for SMA, known as Evrysdi (risdiplam), marked a major milestone, offering a non-invasive, daily oral treatment option for patients of various ages.

The US Food and Drug Administration (FDA) has approved several medications for Spinal Muscular Atrophy (SMA), marking a significant shift from historically palliative care to disease-modifying therapies that can substantially improve patient outcomes. Knowing **what is the FDA approved treatment for SMA** can help patients and their families understand the available options, which target the root genetic cause of the disease.

Since 2016, the U.S. Food and Drug Administration (FDA) has approved three transformative therapies for spinal muscular atrophy (SMA), fundamentally changing the prognosis for many patients. The question of what is the drug of choice for SMA does not have a single answer, as the optimal treatment is a personalized decision based on a patient's age, genetic profile, and disease type.

Before 2016, there were no targeted treatments for spinal muscular atrophy (SMA), and the most severe cases often had a life expectancy of less than two years. Today, revolutionary therapies have dramatically altered the prognosis, with some individuals dubbing one a "miracle drug for SMA". While not a complete cure, these pharmacological advancements address the genetic root cause and offer significant, life-altering improvements for patients, especially when administered early.

Just a decade ago, treatment for spinal muscular atrophy (SMA) was limited to supportive care, but the landscape has been revolutionized by targeted therapies. So, what drugs are used to treat SMA? Today, several FDA-approved medications address the genetic root cause of the disease, offering hope for improved motor function and survival across different patient ages and types.