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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

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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

May 11, 2020May 15, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

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The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. ... Read more

May 11, 2020May 15, 2020

Respiratory Weakness in SMA Most Pronounced in Childhood, Study Finds

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Lung function in people with earlier-onset forms of spinal muscular atrophy (SMA) tends to decline gradually throughout childhood and stabilizes in early adulthood, according to data from a population-based ... Read more

May 8, 2020May 8, 2020

SBMA Seen to Affect Skeletal Muscles First, Not Motor Neurons

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Although a disease of motor neurons, nerve cells of the spinal cord and brainstem, the earliest signs of spinal and bulbar muscular atrophy (SBMA) are in skeletal muscles, according to ... Read more

May 6, 2020May 6, 2020

SMA Treatment and Screening Programs ‘Essential’ in Pandemic, Panel Says

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Continuing treatment for spinal muscular atrophy (SMA) is essential and should not be considered elective, or be delayed or interrupted if possible, despite the healthcare limitations imposed by the COVID-19 ... Read more

May 4, 2020May 15, 2020

Lung Ultrasound Reliable Option to Chest X-rays for Monitoring Children

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Lung ultrasound is a safe and reliable bedside tool to detect atelectasis (partial lung collapse) early in children with neuromuscular diseases, including spinal muscular atrophy (SMA), a study suggests. This radiation-free ... Read more

May 1, 2020May 1, 2020

Mom Writes of Family and Doctor ‘Partnership’ That Led to Baby’s Spinraza Treatment

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A family’s journey through the diagnosis and treatment of their baby girl, born with spinal muscular atrophy type 1 (SMA1), was the focus of a paper written by her ... Read more

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