SMA News Today

August 5, 2020August 6, 2020

Cure SMA Offers Free Medical Alert Bracelet Kits to US Patients

Cure SMA has launched a new program to offer medical alert bracelets to spinal muscular atrophy (SMA) patients in the United States at no cost to the patient, according to ... Read more

August 3, 2020August 3, 2020

New ASAH1 Mutations Linked to Rare Case of SMA and Farber Disease

SMA News Today

A rare instance of a child with both spinal muscular atrophy (SMA) and Farber disease, associated with two new mutations in the ASAH1 gene, is detailed in a case report. Its team ... Read more

July 31, 2020August 3, 2020

Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows

SMA News Today

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which included ... Read more

July 29, 2020July 29, 2020

Plans Advance for Biomarkers Panel to Assess Drug-induced Skeletal Muscle Injury

SMA News Today

A new panel of four safety biomarkers to assess acute drug-induced skeletal muscle injury in Phase 1 clinical trials has received a positive response from the U.S. Food and Drug ... Read more

July 27, 2020July 27, 2020

Blocking Calpain Enzyme Found to Increase SMN Protein in Preclinical Models

SMA News Today

In cell- and mouse-based models of spinal muscular atrophy (SMA), reduced levels of the SMN protein — a hallmark of the disease — altered both the calpain enzyme system and ... Read more

July 24, 2020July 24, 2020

Greatest Loss of Motor Skills at Ages 5 to 13 in SMA Type 2

SMA News Today

People with type 2 spinal muscular atrophy (SMA) show the greatest loss of motor abilities on the Hammersmith Functional Motor Scale Expanded (HFMSE) between the ages of 5 and 13, and ... Read more

July 22, 2020July 22, 2020

Biogen to Launch First Trial Testing Spinraza in Children Previously Given Zolgensma

SMA News Today

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 ... Read more

July 20, 2020July 20, 2020

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

COVID-19 News, SMA News Today

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 ... Read more

July 17, 2020July 17, 2020

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

SMA News Today

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 ... Read more

July 15, 2020July 15, 2020

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

SMA News Today

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. ... Read more

July 13, 2020July 13, 2020

Iowa Adds SMA as Pilot Program for Newborn Screening

SMA News Today

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 ... Read more

July 10, 2020July 10, 2020

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

SMA News Today

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 ... Read more

July 8, 2020July 8, 2020

Illinois Expands Newborn Screening to Include SMA

SMA News Today

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 ... Read more

July 6, 2020July 6, 2020

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

SMA News Today

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. ... Read more

July 1, 2020July 1, 2020

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

SMA News Today

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 ... Read more