Despite Favorable Safety, Olesoxime Falls Short Among Efficacy Markers for SMA
December 10th 2020Long-term follow-up of the treatment showed that while it has a favorable safety profile, the treatment does not offer significant efficacy for patients with spinal muscular atrophy (SMA) type 2 and non-ambulant type 3.
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Population Carrier Screening Increases Prenatal SMA Diagnoses, Reduces SMA Births
December 7th 2020Between the first introduction of population carrier screening in 2008 and its expansion to the full Israeli population in 2013, the mean rate of prenatal diagnoses per year was 4.66, and this rose to 7.75 between 2014 and 2017, a 66% increase.
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Case Report Shows Neuropathological, Molecular Findings of SMA Type 3 With Superficial Siderosis
November 28th 2020Researchers have published their postmortem report on a case of spinal muscular atrophy type 3 complicated by superficial siderosis, offering details on their neuropathological and molecular findings.
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Analysis Shows Utility of Tool to Detect Motor Function Change in SMA
October 17th 2020Analyzing data from a phase 2 study of patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA,) researchers found that the assessment is a valid, reliable, and responsive assessment of motor function ability for these patients.
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Survey Captures Baseline Experience of Patients With SMA
October 9th 2020A recent study about the impact of spinal muscular atrophy (SMA) on patients and caregivers may help to illustrate the impact of future improvements as new therapies take hold, but more targeted SMA quality-of life-outcome measures may be needed to fully the changes.
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New Research Offers Key Safety, Early Efficacy Data on Zolgensma in SMA
September 25th 2020A year after the FDA approved Zolgensma for the treatment of spinal muscular atrophy in children aged under 2 years without end-stage weakness, researchers shared safety and early efficacy data from the first 21 children treated with the gene therapy in Ohio.
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