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July 20, 2020July 20, 2020

Study of Physical Activity by Patients in UK Under COVID-19 Opens

News

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 ... Read more

July 17, 2020July 20, 2020

Spinraza Eases Fatigue in Adults With SMA But Benefit Wanes, Study Finds

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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 ... Read more

July 15, 2020July 16, 2020

Guidance on Zolgensma in Europe Notes Lack of ‘Evidence’ for Broad Use

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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 ... Read more

July 13, 2020July 13, 2020

Iowa Adds SMA as Pilot Program for Newborn Screening

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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 ... Read more

July 10, 2020July 10, 2020

Asuragen’s Diagnostic Test for SMA Approved for Use in Europe

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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 ... Read more

July 9, 2020July 9, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

News, syndicated

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 ... Read more

July 8, 2020July 8, 2020

Illinois Expands Newborn Screening to Include SMA

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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 ... Read more

July 6, 2020July 6, 2020

SMN Protein Levels in Blood May Mark SMA Severity, Help Guide Treatment

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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 ... Read more

July 1, 2020July 1, 2020

New Glia Cell Markers May Provide Insight Into Neuromuscular Diseases, Study Says

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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 ... Read more

June 29, 2020July 10, 2020

‘Significant’ Limits to Treatment Seen for SMA Type 0 Baby in Case Report

News

A baby with spinal muscular atrophy (SMA) type 0 treated with both Spinraza (nusinersen) and Zolgensma is showing benefits relative to the natural course of this most severe disease form, but ... Read more

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