Tag: Onasemnogene abeparvovec

Affecting approximately 1 in every 10,000 live births, spinal muscular atrophy (SMA) is a leading genetic cause of infant death. Groundbreaking advances in medicine now offer life-changing therapeutic options, and the question, "What is the injection for spinal muscular atrophy?" points to key treatments like Nusinersen (Spinraza) and Onasemnogene Abeparvovec (Zolgensma).

In untreated cases, Spinal Muscular Atrophy (SMA) type 1, the most common and severe form, results in a low survival rate beyond the age of two. Zolgensma, a groundbreaking gene replacement therapy, works by delivering a healthy copy of the SMN1 gene to treat the underlying genetic cause of this devastating disease.

The treatment landscape for Spinal Muscular Atrophy (SMA) has undergone a dramatic transformation in recent years, shifting from purely supportive care to advanced, genetically targeted therapies. In response to the question, **what medication is used to treat SMA?**, three primary FDA-approved options—Nusinersen (Spinraza), Onasemnogene abeparvovec (Zolgensma), and Risdiplam (Evrysdi)—now offer a range of approaches to address the root genetic cause of the disease. This breakthrough in pharmacology has fundamentally changed the prognosis for many patients.

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.