Articles, Newsletters, Podcasts, and Video

Spinraza Stabilizes Motor Function for Adults With SMA, Study Reports

By Marisa Wexler MS / 2022-05-13
Posted in

Treatment with Spinraza (nusinersen) modestly improved muscle strength and stabilized motor function for adults with spinal muscular atrophy (SMA) types 2 or 3, according to

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Girl, 16, Becomes 1st SMA Patient to Undergo Hip Replacement Surgery

By Vanda Pinto / 2022-05-02
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A surgeon at UC Davis Health has successfully performed the first hip replacement in a person — in this case, a 16-year-old girl — with

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#AAN2022 – Spinraza Showing Safety in Children With Needs After Zolgensma

By Marta Figueiredo PhD / 2022-04-29
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Treatment with Spinraza (nusinersen) appears safe in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma, early safety data from

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Who Am I? Embracing My SMA Advocacy

By Kevin Schaefer / 2022-04-26
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I was at a friend’s wedding a few months ago when I pondered the question of how to properly introduce myself. Typically, I give the

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Combining Therapies May Improve Efficacy in SMA: Mouse Model

By Steve Bryson PhD / 2022-04-22
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A combined spinal muscular atrophy (SMA) therapy — one incorporating two treatments targeting different disease processes related to the SMN protein — showed improved efficacy

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Spinraza Most Effective in Younger Type 1 Children: Real-world Study

By Steve Bryson PhD / 2022-04-15
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In a real-world study involving more than 40 Swiss patients with spinal muscular atrophy (SMA), Spinraza (nusinersen) was found to effectively improve motor function — particularly among

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Groups Join Forces to Push for SMA Newborn Screening in Scotland

By Mary Chapman / 2022-04-13
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As part of ongoing efforts to have all newborns in the U.K. screened for spinal muscular atrophy (SMA), Muscular Dystrophy UK (MDUK) recently met with

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Celebrating My 1st Evrysdi-versary and Looking to the Future

By Brianna Albers / 2022-04-11
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My first Evrysdi-versary passed without incident. Ironic, considering how much time and effort I put into acquiring the medication. A disease-modifying treatment (DMT) for SMA

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2 UK Nonprofits Fund PhD Student to Study SMA Mechanisms, Treatments

By Lindsey Shapiro PhD / 2022-04-04
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A couple of nonprofits in the U.K. will jointly sponsor a PhD student’s research into the mechanisms underlying nerve degeneration and potential treatment approaches for

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‘Garbage Disposal’ Protein May Help in Predicting Response to Spinraza

By Marisa Wexler MS / 2022-04-01
Posted in

In children with spinal muscular atrophy (SMA) who begin treatment with Spinraza (nusinersen) after they are 2 months old, levels of a protein called cathepsin

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