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October 18, 2019October 18, 2019

SMA Patients May Benefit from Low-fat Diet, Mouse Study Suggests

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Adhering to a low-fat diet may be beneficial for people with spinal muscular atrophy (SMA), a mouse study suggests. Mice fed on low-fat diets lived longer, and showed reduced ... Read more

October 16, 2019October 16, 2019

SMN Protein Study in Fetuses and Children Emphasizes Importance of Early SMA Treatment, Newborn Screening

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Levels of the survival motor neuron (SMN) protein — which are significantly low in spinal muscular atrophy (SMA) patients — naturally rise during gestation and then fall in the three ... Read more

October 14, 2019October 14, 2019

Spinraza Improves or Stabilizes Motor, Lung Function in SMA Type 3 Adults, German Study Suggests

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Spinraza (nusinersen) improves or stabilizes motor and lung functions in adults with spinal muscular atrophy (SMA) type 3, an observational German study suggests. The findings, which are significant compared with the disease’s ... Read more

October 11, 2019October 18, 2019

Zolgensma Produces Promising Results in Young Children with Type 2 SMA, Early Trial Data Show

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A single injection of Zolgensma into the spinal canal (intrathecal) can provide a clinically meaningful response in motor function gains among young children, 2 to 5 years old, who ... Read more

October 9, 2019October 10, 2019

Early Data Support SRK-015 Development for SMA Types 2–3, Scholar Rock Says

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SRK-015, an experimental treatment for spinal muscular atrophy (SMA), continues to produce promising results in preclinical studies and in a Phase 1 trial of healthy volunteers, its developer, Scholar Rock, ... Read more

October 7, 2019October 10, 2019

Risdiplam Continues to Show Survival, Motor Function Improvements, According to Trial Results

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Risdiplam continues to show promise as a safe and effective therapy for people with spinal muscular atrophy (SMA) types 1, 2 and 3, according to the latest results from ongoing clinical trials. Data from the ... Read more

October 4, 2019October 10, 2019

Researchers Develop Fast, Low-Cost, and Non-invasive Prenatal Screening Test for SMA

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Chinese researchers have developed a fast, low-cost, and non-invasive maternal blood test for prenatal screening of spinal muscular atrophy (SMA). This method may be a safer alternative to traditional ... Read more

October 2, 2019October 10, 2019

Scientists Develop New Method to Produce High Quantities of Viral Vectors for Gene Therapy

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Scientists have developed a fast, efficient and economical method to create viral delivery vectors used in gene therapy to deliver modified versions of genes to treat disorders caused by ... Read more

September 30, 2019October 10, 2019

SMA Type 1 Patients May Be at Greater Risk of Kidney Dysfunction, Study Finds

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People with spinal muscular atrophy (SMA) type 1 may have an increased risk of impaired kidney function, a small study suggests. The study’s findings highlight the need to further ... Read more

September 27, 2019October 2, 2019

In Historic Move, Israeli Insurer to Cover Cost of Zolgensma for 2 SMA Babies

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In an unprecedented decision reached Sept. 26 on the eve of Rosh Hashana — the Jewish New Year — Israel’s top health insurer, Clalit, said it would cover the ... Read more

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