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Biogen has announced plans to launch a Phase 4 clinical trial evaluating the benefits of Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who were previously treated with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi). The company expects to enroll the first patients…
The University of Birmingham has begun a study to gauge the impact of the COVID-19 pandemic on adults in the United Kingdom (UK) with neurological diseases, such as spinal muscular atrophy (SMA), that affect mobility. The series of online surveys, to be completed over 12 months,…
Spinraza significantly eased fatigue in adults with spinal muscular atrophy (SMA) after six months of treatment, as measured by the patient-reported Fatigue Severity Scale (FSS), a small study found. But this relief was no longer statistically significant after 10 months,…
Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional SMA types, according to a consensus statement from a panel of European experts. The statement covers 11 points, and highlights that clinical trials of Zolgensma to date involved…
The Iowa Department of Public Health has begun a pilot effort to screen newborns for spinal muscular atrophy (SMA). Babies born in Iowa are now being screened for more than 40 inherited disorders, with a goal of identifying conditions that, if caught early, can be treated or…
Asuragen’s diagnostic test for the analysis of genes associated with spinal muscular atrophy (SMA) — called AmplideX SMA Plus Kit — was given the European CE mark, approving it for commercial use, the company announced. The designation means the AmplideX kit complies with the…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just…
The Illinois Department of Public Health (IDPH) now includes spinal muscular atrophy (SMA) as part of its newborn screening effort. The addition of SMA — the 49th disorder in the state’s screening program — means that every baby born in Illinois will be tested for the autosomal…
Levels of the survival motor neuron (SMN) protein in the blood of people with spinal muscular atrophy (SMA) shows promise as a biomarker for predicting disease severity, a study reports. Specifically, SMN levels correlate with the severity of motor-neuron loss — denervation — and with the number…
Perisynaptic Schwann cells — a specialized type of cell that helps facilitate communication between neurons and muscle cells — express specific molecular markers, which could make it easier to identify and investigate these cells, a new study has found. Better understanding these cells’ biology and function…
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