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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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This month has seen a surge of publications focused on SMA treatments, with a trend towards optimizing various aspects of nusinersen treatment. Research has also continued in areas related to molecular mechanisms of the disease, clinical manifestations, and management protocols. Here’s a roundup of the latest in SMA research…
February wrapped up with a surge of data on SMA, including novel investigations into how combinatorial therapies may provide new advantages to those with the disease. More information on how nusinersen impacts patients has also become available, as have new findings related to newborn screening and the genetics underlying SMA.
Every patient with SMA has different abilities and deficits as well as different ways of transportation. Most are unable to walk independently, therefore require assistance of some sort. Michael, our patient with SMA, has a power chair that he uses to navigate around. He has his controls on the right…
Research on SMA continues to forge ahead in 2020, with new results in from Roche’s latest investigation into its oral SMA drug, risdiplam. New tools for screening are being revealed as legislative changes are pushing more and more toward requiring infants born in the U.S. to be screened for SMA.
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