News

December 30, 2020

Informal SMA Caregivers Surveyed in Europe Report High Daily Burden

News

Non-professional caregivers who care for children with spinal muscular atrophy (SMA) carry a high daily burden that negatively affects certain aspects of their lives, according to a questionnaire study ... Read more

December 21, 2020December 21, 2020

Zolgensma Linked in 3 Cases to Serious But Treatable Blood Disorder

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Some children with spinal muscular atrophy (SMA) may develop thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — following treatment with Zolgensma, a study reports. A ... Read more

December 18, 2020December 18, 2020

Zolgensma Approved to Treat Young SMA Patients in Canada

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Health Canada has approved Novartis‘ gene therapy Zolgensma (onasemnogene abeparvovec) to treat pediatric spinal muscular atrophy (SMA) patients who have three or fewer copies of the SMN2 gene, or with infantile-onset ... Read more

December 16, 2020December 16, 2020

Paramedian Injection Approach Found to Ease Spinraza Administration

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Administering Spinraza (nusinersen) via a particular method of injection, called the paramedian approach, can shorten procedure times and reduce the occurrence of adverse events for people with spinal muscular ... Read more

December 14, 2020December 14, 2020

Financial Burden of SMA Much Higher Than Other Conditions, Analysis Shows

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Direct medical costs associated with spinal muscular atrophy (SMA) are more than 50 times higher than those of matched control patients without SMA, according to a healthcare claims analysis. ... Read more

December 11, 2020December 11, 2020

Measuring Neurological Impact of Polio Proves Useful in SMA, Study Reports

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Researchers have found changes in the electrical activity between the brain and muscles in adults who had childhood polio — an infection that affects the same motor neurons as ... Read more

December 9, 2020December 9, 2020

Genetic Analysis of Families Finds Novel Mutations in SMA

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Genetic analysis of 28 families with a member affected by spinal muscular atrophy (SMA) identified novel mutations in the SMN1 gene and different genes widely prevalent in SMA patients ... Read more

December 7, 2020December 7, 2020

Zolgensma-associated Liver Abnormalities Common but Manageable, Study Shows

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Most children with spinal muscular atrophy (SMA) show an abnormal increase in the levels of liver enzymes — suggestive of liver damage — following treatment with Zolgensma, according to a study ... Read more

December 4, 2020December 4, 2020

MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

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The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention(CDC) to recommend that people living with neuromuscular diseases (NMDs) have early access to any ... Read more

December 4, 2020December 4, 2020

Neurological Alliance Report Calls for Better Treatment, Care for UK Patients

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A Neurological Alliance report is calling for improved treatment and care for the 150,000 U.K. residents living with rare neurological conditions such as spinal muscular atrophy (SMA). The report ... Read more