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SMA News Today HCP is the web’s only dedicated resource for serving the healthcare community with information and insights about treating spinal muscular atrophy.

01/12/2023

Despite its well-reported benefits in motor function, the gene therapy Zolgensma may not be as effective at preventing or easing breathing problems at night in young children with spinal muscular atrophy, according to a small real-world study. https://buff.ly/3ZkyfoP

Breathing problems at night may be common in young children with SMA after treatment with Zolgensma gene therapy, a real-world study found.

01/09/2023

A significant proportion of people with spinal muscular atrophy do not remain on Spinraza or receive treatment injections as prescribed, according to a U.S. insurance claims analysis. https://buff.ly/3IE8V7d

Few spinal muscular atrophy patients stick to their prescribed Spinraza treatment regimen, a new claims analysis using real-world data found.

01/05/2023

A technique called bioelectrical impedance analysis can be used to measure body composition in people with type 2 or 3 spinal muscular atrophy, a new study reports. https://buff.ly/3Vj7Qoc

Bioelectrical impedance analysis (BIA) can be an easier method to measure body composition in people with type 2 or 3 SMA, a study finds.

01/02/2023

The U.S. Food and Drug Administration (FDA) has given NMD Pharma the green light to conduct a Phase 2 trial of its oral therapy NMD670 in people with spinal muscular atrophy (SMA) type 3. https://buff.ly/3C08CQa

The U.S. Food and Drug Administration has cleared a Phase 2 clinical trial of NMD Pharma's oral therapy NMD670 for patients with SMA type 3.

12/29/2022

The elevated production of the cell-death-related protein c-Fos was closely linked to the death of motor neurons in two mouse models of spinal muscular atrophy (SMA), a study showed. Motor neurons are the muscle-controlling nerve cells that are lost in people with SMA. https://buff.ly/3USdr4o

The elevated production of the cell-death protein c-Fos was linked to the death of motor neurons in two spinal muscular atrophy mouse models.

12/26/2022

People with spinal muscular atrophy (SMA) types 2 or 3 who start treatment with Spinraza (nusinersen) and show less muscle damage on MRI scans are more likely to see motor function improvements, a new study reports. https://buff.ly/3HaOqhO

Spinal muscular atrophy patients taking Spinraza who show less muscle damage on MRI scans are more likely to have motor function improvements.

12/22/2022

There are more than 1,200 people with spinal muscular atrophy living in Italy, and the vast majority are on disease-modifying treatment, according to a new study. https://buff.ly/3V67OzX

There are more than 1,200 people with SMA living in Italy, and the vast majority are on disease-modifying treatment.

12/20/2022

UNITY Screen, a noninvasive prenatal blood test, can provide an accurate personalized risk assessment for hereditary diseases such as spinal muscular atrophy (SMA) in fetuses, according to a recent study https://buff.ly/3j5fZyX

UNITY Screen, a non-invasive prenatal blood test, accurately assessed risk for hereditary diseases in fetuses, such as SMA

12/15/2022

The elevated production of the cell-death-related protein c-Fos was closely linked to the death of motor neurons in two mouse models of spinal muscular atrophy, a study showed. Motor neurons are the muscle-controlling nerve cells that are lost in people with SMA. https://buff.ly/3USdr4o

The elevated production of the cell-death protein c-Fos was linked to the death of motor neurons in two spinal muscular atrophy mouse models.

12/08/2022

Severe scoliosis — an abnormal curvature of the spine — was significantly associated with older age and limited motor abilities in children with spinal muscular atrophy type 2 who had not received disease-modifying therapies, a study showed. https://buff.ly/3VrCqNb

More severe scoliosis was associated with older age and lower motor skills in children with SMA type 2 not on disease-modifying therapies.

12/06/2022

A new system that contains a gene-modulating therapy attached to an antibody that enables crossing into the nervous system improved survival outcomes in a mouse model of severe spinal muscular atrophy (SMA), a new study reports. https://buff.ly/3BfYmmj

A gene-modulating therapy attached to an antibody that allows crossing into the nervous system improved survival in mice with severe SMA.

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