SMA News Today

June 3, 2020June 3, 2020

Too Little Known About Care Needs of Adults with SMA, Study Says

SMA News Today

A better understanding of the needs of adults with spinal muscular atrophy (SMA), best ensuring for their mental and social — as well as physical — well-being, is necessary to guide future ... Read more

June 1, 2020June 1, 2020

Image-guided Methods May Help Lower Complications in Administering Spinraza

SMA News Today

Adopting image-guided techniques may help physicians lower the risks of complications associated with the administration of Spinraza (nusinersen) in patients with spinal muscular atrophy (SMA), a study has found. Physicians ... Read more

May 29, 2020May 29, 2020

Plant Compound, Berberine, Seen to Clear Protein Clumps That Mark SBMA

SMA News Today

Berberine, a plant-derived compound often used in Chinese medicine, is able to clear the toxic protein clumps found in neurodegenerative diseases such as spinal and bulbar muscular atrophy (SBMA), amyotrophic lateral ... Read more

May 27, 2020May 27, 2020

SMA Less Common Than Expected Among Newborns Screened in NY

SMA News Today

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 of ... Read more

May 22, 2020May 22, 2020

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

SMA News Today

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 suggests. ... Read more

May 20, 2020May 20, 2020

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

COVID-19 News, SMA News Today

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 to ... Read more

May 19, 2020May 19, 2020

European Commission Grants Conditional Approval to Zolgensma for Treating SMA

SMA News Today

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 for ... Read more

May 18, 2020May 18, 2020

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

SMA News Today

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 study, ... Read more

May 15, 2020May 15, 2020

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

COVID-19 News, SMA News Today

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 COVID-19 ... Read more

May 13, 2020May 13, 2020

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

SMA News Today

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 also ... Read more

May 11, 2020May 11, 2020

Respiratory Weakness in SMA Most Pronounced in Childhood, Study Finds

SMA News Today

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 natural ... Read more

May 8, 2020May 8, 2020

SBMA Seen to Affect Skeletal Muscles First, Not Motor Neurons

SMA News Today

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 data ... Read more

May 6, 2020May 6, 2020

Ella’s Got Talent

SMA News Today

Ella is a social person. She loves developing relationships with others and is always up for a family night. She builds delicate yet sturdy friendships with people and navigates these ... Read more

May 6, 2020May 8, 2020

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

COVID-19 News, SMA News Today

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 pandemic.   ... Read more

May 5, 2020May 8, 2020

SMA Humor Writes Itself

COVID-19 Columns, SMA News Today

The comedian Brad Williams has a great one-liner about writing jokes. Williams’ observational humor is predominantly rooted in his experiences as a man born with achondroplasia, a type of dwarfism. ... Read more