Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show
Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function and safety, new interim data from the STRONG trial in spinal muscular atrophy (SMA) type 2 patients up to age 5 show. These findings from the open-label...
SMA in Mid-March: Clinical Practice Guidelines, Walking and Weakness in Children & More
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...
Lake-Breaking SMA Treatment Insights You May Have Missed in February
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...
SMA in Late February: Roche’s Sunfish Trial, Intrathecal Nusinersen Treatment & More
Perhaps the biggest SMA news this month has been the success of Roche’s Sunfish trial, but fundamental research, such as those into the mechanisms of the disease and the implications of certain interventions, continue. Below is a roundup of the latest news and...
SMA HCP Roundup, mid-February: X-linked infantile SMA, Risdiplam News & More
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...HCP — SMA News Today
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