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May 27, 2020May 27, 2020

SMA Less Common Than Expected Among Newborns Screened in NY

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The frequency with which spinal muscular atrophy (SMA) is found in newborns may be lower than previously thought, according to one-year data from an SMA screening program in the state ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 22, 2020May 22, 2020

Heart Problems in SMA May Be Tied to Calcium Dysregulation, Study Suggests

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Heart problems associated with spinal muscular atrophy (SMA) may be caused partially by calcium dysregulation in heart muscle cells in the absence of the survival motor neuron (SMN) protein, a study ... Read more

May 21, 2020May 21, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

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Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s ... Read more

May 20, 2020May 20, 2020

Spinraza Shows Sustained Efficacy Over Years of Use, SHINE Data Find

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Years of treatment with Spinraza (nusinersen) show sustained effectiveness, from motor skill gains to disease stabilization, and continued safety across a broad range of spinal muscular atrophy (SMA) patients, from toddlers ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 19, 2020May 19, 2020

European Commission Grants Conditional Approval to Zolgensma for Treating SMA

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The European Commission (EC) has granted conditional approval to Zolgensma (onasemnogene abeparvovec) for treating patients with spinal muscular atrophy (SMA) and a clinical diagnosis of type 1 SMA, or ... Read more

May 18, 2020May 18, 2020

Feeding Issues Still Common Among SMA Type 1 Infants, Despite New Therapy Options

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Despite recent treatment advances, feeding difficulties are still one of the most common complications for infants with spinal muscular atrophy (SMA) type 1, an observational study has found. The ... Read more

May 15, 2020May 15, 2020

6-month Analysis of SMA Patients in TOPAZ Trial Delayed to Year’s End

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A planned six-month interim analysis of a Phase 2 study of SRK-015 in children and adults with spinal muscular atrophy (SMA) will be delayed by about three months due to the ... Read more

May 13, 2020May 15, 2020

Spinraza Meets Most Therapeutic Expectations of Adult SMA Patients, Real-World Study Shows

News

Spinraza (nusinersen) improves muscle strength, endurance, and independence in adults with spinal muscular atrophy (SMA), meeting most of their positive expectations, a small, real-world study in Germany has shown. The data ... Read more

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