SMA type 2 in a 4-year-old – what new options are there besides Spinraza?
Hello everyone. My nephew was diagnosed with SMA type 2 at 1.5 years old. He’s on Spinraza injections now, can sit with support and crawls a little, but progress is very slow. We keep hearing about gene therapy (Zolgensma) and some clinics abroad offering additional stem cell approaches for SMA. Has anyone looked into this seriously? Is it worth considering for a child who already started nusinersen, or is it too late/risky?
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From what families in our local SMA group share, Zolgensma is usually done before age 2, but some clinics offer combined or follow-up protocols even later - especially stem cell-based ones aimed at supporting motor neuron survival and muscle function. Results aren’t dramatic like in very young kids, but several parents report small but steady gains: better head control, less fatigue, sometimes improved swallowing.
One quite detailed story from a family whose child was 3.5–4 years old at the start (with before/after motor scale scores) is here https://stemplus.clinic/disease-treatment/sma/ - it’s not promising a cure, just showing realistic steps and changes over 12–18 months. Of course, every case is individual, and you’d need to consult a pediatric neurologist who knows SMA well before any decision.