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There are many different modalities and activities that have been successful in progressing Michael, our patient with SMA, towards reaching his goals of less pain during activities as well as increased independence. Over the past four years, we have attempted many different techniques with Michael and have noted the ones…
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Since our last roundup, there has been continued focus in the spinal muscular atrophy (SMA) literature how best to treat the disease. There also continues to be research geared toward understanding the biology of the disease, as well as on SMA management, what it is like to live with the…
[two_third_last] If you are working with a patient with SMA, you will most likely find that they are in their wheelchair full time. This makes sitting balance very important for many reasons. If they have good sitting balance, this will make traveling up and down ramps easier and more…
Much of the most recent research regarding spinal muscular atrophy (SMA) has focused on understanding the mechanisms mediating the disease as well as the clinical value of different pharmaceutical interventions. There have been several reviews on the latter topic, with a continuing emphasis on nusinersen, the first drug approved to…
Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug Administration (FDA) announced today, issuing an historic decision.
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