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. The upcoming clinical trial aims to evaluate the efficacy, safety, and tolerability of…
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There are more than 1,200 people with spinal muscular atrophy (SMA) living in Italy, and the vast majority are on disease-modifying treatment, according to a new study. SMA types 2 and 3 are the most common forms of the disease in the country, but the prevalence…
The use of long-term breathing help, or ventilation, has markedly increased among children with spinal muscular atrophy (SMA) type 1 and other conditions since 2008, according to a survey of ventilation centers in the U.K. This increase coincided with the expanded use of various types of non-invasive masks as…
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. Offered by BillionToOne in the U.S., the single-gene noninvasive prenatal test (sgNIPT) included in UNITY Screen requires…
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. Although c-Fos didn’t directly cause the…
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. The finding “underscores the importance of early diagnosis and treatment to obtain…
Severe scoliosis — an abnormal curvature of the spine — was significantly associated with older age and limited motor abilities in children with spinal muscular atrophy (SMA) type 2 who had not received disease-modifying therapies, a study showed. These findings establish characteristics of untreated scoliosis progression on SMA type…
Without disease-modifying treatment (DMT), spinal muscular atrophy (SMA) carries high mortality rates and steep healthcare costs, a study in Hong Kong reported. Not surprisingly, the greatest loss of life and highest costs were observed in children with SMA type 1, one of this disease’s most severe forms with…
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. The study, “Antibody-oligonucleotide conjugate achieves central nervous system delivery in…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was safe and effective in a baby girl with spinal muscular atrophy (SMA) type 1 who was previously on Evrysdi (risdiplam) for about three months, a case study reports. This likely represents the first report of safety and efficacy outcomes of…
