SMA News Today

January 15, 2021January 15, 2021

Online for 2021, Team Cure SMA Race Series Planning Variety of Events

The COVID-19 pandemic has forced many activities to be online only, but in the case of this year’s Team Cure SMA endurance race series, the limitation means more people can ... Read more

January 12, 2021January 13, 2021

Spinraza Trial Begins Treating Children Who Failed to Respond to Gene Therapy

SMA News Today

The first patient has been dosed in a Phase 4 trial exploring the safety and potential benefits of Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) who failed to ... Read more

January 8, 2021January 8, 2021

New Cure SMA Webinar Series to Focus on Health, Wellness

COVID-19 News, SMA News Today

Cure SMA has developed a new three-part webinar series that delves into health and wellness topics pertinent to the spinal muscular atrophy (SMA) community. The three monthly installments of the ... Read more

January 6, 2021January 6, 2021

Higher-dose Spinraza Trial Now Enrolling Part B After No Safety Issues Found

SMA News Today

After finding no safety concerns in Part A of the DEVOTE trial, which is testing higher doses of Spinraza (nusinersen) among people of all ages with spinal muscular atrophy (SMA), investigators ... Read more

January 4, 2021January 4, 2021

Informal SMA Caregivers Surveyed in Europe Report High Daily Burden

SMA News Today

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 conducted ... Read more

December 21, 2020December 21, 2020

Zolgensma Linked in 3 Cases to Serious But Treatable Blood Disorder

SMA News Today

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 serious ... Read more

December 18, 2020December 18, 2020

Zolgensma Approved to Treat Young SMA Patients in Canada

SMA News Today

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 disease. ... Read more

December 16, 2020December 16, 2020

Paramedian Injection Approach Found to Ease Spinraza Administration

SMA News Today

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 atrophy ... Read more

December 14, 2020December 14, 2020

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

SMA News Today

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. The ... Read more

December 11, 2020December 11, 2020

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

SMA News Today

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 those ... Read more

December 9, 2020December 9, 2020

Genetic Analysis of Families Finds Novel Mutations in SMA

SMA News Today

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 not ... Read more

December 7, 2020December 7, 2020

Zolgensma-associated Liver Abnormalities Common but Manageable, Study Shows

SMA News Today

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 by ... Read more

December 4, 2020December 4, 2020

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

COVID-19 News, SMA News Today

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 federally ... Read more

December 4, 2020December 4, 2020

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

COVID-19 News, SMA News Today

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 presents ... Read more