spinal muscular atrophy (sma)

What are the Treatment Options for SMA?

While there is no cure for SMA, recent advances have led to the development of treatments that can improve outcomes. Key treatments include:
Nusinersen (Spinraza): An antisense oligonucleotide therapy that increases the production of the SMN protein.
Onasemnogene abeparvovec-xioi (Zolgensma): A gene therapy that delivers a functional copy of the SMN1 gene.
Risdiplam (Evrysdi): An oral medication that increases SMN protein levels.
Supportive care is also crucial and includes respiratory support, nutritional support, physical therapy, and orthopedic interventions.

Frequently asked queries:

What is Spinal Muscular Atrophy (SMA)?
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