One problem seen with SMA is that its window of opportunity is quite short, especially in infants with type 1 SMA. This period is not exactly known; however, at some point, even if we intervene using stem cell therapy, clinical benefit might not be optimum – or might be temporary to the duration of stem cell therapy injections. This has been suggested by one study that noted that the benefits seen in their type 1 SMA patients – who were all older than 7 months old – had temporary benefits despite them showing significant improvement during the period of therapy.

They have therefore advised that earlier stem cell therapy might be a solution; prior to permanent damage to the neurons of children with SMA. Of course, this treatment window differs according to the SMA subtype your child has and the severity of their symptoms, and the reason most trials done on patients with SMA showed partial benefits is possible that they recruited older infants with the more severe type 1 SMA type who had probably acquired permanent neuron damage due to their older age (9, 10).

Therefore, for people with type 1 SMA an age prior to 4 to 6 months might be the optimum window for treatment.

People with less severe forms might however have benefits long after this period depending on the severity of the symptoms. This is why we recommend consulting our specialists prior to treatment in order to estimate the degree of benefit that can be obtained from stem cell therapy.

We can generally say that the earlier stem cell therapy is initiated, the better the expected results are.