Study Addresses Dilemmas Regarding Newborn Screening and SMA Treatment

Study Addresses Dilemmas Regarding Newborn Screening and SMA Treatment

As newborn screening for spinal muscular atrophy (SMA) becomes more common, ethical dilemmas will arise about the best treatment strategy for individuals with four copies of the SMN2 gene, a new study contends. The study, “Infants Diagnosed with Spinal Muscular...
Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show

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

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

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 HCP Roundup, mid-February: X-linked infantile SMA, Risdiplam News & More

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...