Spinal Muscular Atrophy (SMA) is a genetic disease that affects the central nervous system and muscles. Missing or mutated genes result in a deficiency of a protein (SMN) that motor neurons in the spinal cord need to survive. As motor neurons are lost, the muscles in the body that they control become weak and cannot be used.
TREATMENT OPTIONS
Spinraza (nusinersen) : This medication boosts the production of an important protein, known as sensor motor neuron (SMN) protein². It is administered via injections into the spine, given every four months after an initial loading. Costing: Requires treatment every four months. Lifetime Treatment cost may reach upto INR 24 Cr+*
Evrysdi (risdiplam): This is a daily therapy that can be taken orally or through a feeding tube. Costing: Requires treatment every day. Lifetime Treatment cost may reach upto INR 50 Cr+*
Zolgensma: This therapy is approved to treat SMA in children under 2 years old. Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein Costing: One time Treatment. Lifetime Treatment cost may reach upto INR 17.5 Cr*
Please note above calculations are rough estimate considering average lifespan of human up to 75 years
WHY ZOLGENSMA ?
Zolgensma is made up of a new, working SMN gene that is placed inside a delivery vehicle called a vector. This allows the cells—particularly motor neuron cells—to produce SMN protein.
Zolgensma works by providing a new copy of the SMN gene that produces the SMN protein. Common side effects include vomiting and elevated liver enzymes, while more severe reactions involve liver issues and low platelet count. Shivanshi is a well-preserved baby who would do better with Zolgensma and that would be the best choice for her with better improvements at her current stage. This treatment is best-suited as per the recommendation by Doctors of Baptist Hospital, Bengaluru & AIIMS Hospital, New Delhi