Articles, Newsletters, Podcasts, and Video

Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor Problems

By Marta Figueiredo PhD / 2022-01-17
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Despite its well-reported benefits in motor function, Spinraza (nusinersen) may not be as effective at preventing or easing nonmotor complications, such as facial deformities, feeding

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US Medical Data Show Higher Mortality in SMA Patients Before DMT Era

By Marta Figueiredo PhD / 2022-01-10
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Before the emergence of Spinraza (nusinersen) — the first disease-modifying therapy (DMT) approved for spinal muscular atrophy (SMA) — patients of all ages and types

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Biogen Acquires New Experimental SMA Therapy BIIB115 From Ionis

By Marisa Wexler MS / 2022-01-04
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Biogen has exercised an option to acquire BIIB115, an investigational treatment for spinal muscular atrophy (SMA), from Ionis Pharmaceuticals.

SMA is a rare genetic condition

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Top 10 SMA Stories of 2021

By Marta Figueiredo PhD / 2022-01-03
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SMA News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to spinal muscular atrophy (SMA) throughout 2021.

As

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Spinraza Plus Zolgensma Offers Little Extra Benefit in Type 1, Study Suggests

By Marisa Wexler MS / 2021-12-19
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Combined treatment with Spinraza and Zolgensma does not markedly improve motor function or breathing ability in children with spinal muscular atrophy (SMA) type 1, a

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Spine Surgery Effective in SMA Type 1 Children, But Not Without Risks

By Marta Figueiredo PhD / 2021-12-17
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Surgery to correct kyphoscoliosis — a combination of two types of abnormal curvature of the spine — allows the successful delivery of Spinraza (nusinersen) into the

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Scholar Rock Shares Design of Phase 3 SAPPHIRE Trial of Apitegromab

By Marta Figueiredo PhD / 2021-12-10
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Scholar Rock announced the full design of the upcoming Phase 3 clinical trial testing apitegromab, its experimental muscle-directed therapy for spinal muscular atrophy, in children and

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As an Adult With SMA, I Need Access to Evrysdi, Too

By Sherry Toh / 2021-12-08
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Let’s start this story about Evrysdi (risdiplam) from the beginning.

About a year ago, the chronic neuropathy in my scalp that had been bothering me

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Spinraza May Restore Development of Motor Neurons in SMA Children

By Margarida Maia PhD / 2021-12-08
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Spinraza (nusinersen) may restore the normal development of motor neurons — the nerve cells responsible for making muscle cells work — in children with spinal muscular atrophy (SMA),

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Funding Bolsters Effort to Bring Newborn Screening to Eastern Canada

By Vanda Pinto / 2021-12-03
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A CA$414,000 grant was awarded to the Maritimes by Muscular Dystrophy Canada (MDC) and Novartis Pharmaceuticals Canada to further support the implementation of newborn screening

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