Vivonoetics is partnering with Great Lakes NeuroTechnologies to provide advanced respiratory assessment to the Roche FIREFISH Phase 2 clinical trial in infants with type 1 spinal muscular atrophy type 1 (SMA-1). Vivonoetics is the developer of VivoSense data analysis and software and wearable monitoring devices that have supported research…
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RTI International has developed a groundbreaking program of free genetic-disease testing for newborns, with the initial screenings being for the spinal muscular atrophy (SMA) and fragile X syndrome. Although most American newborns are tested for a number of genetic diseases, screenings are not done for others because they are too…
Researchers conducted a questionnaire-based study to learn more about pregnancy and delivery in women with spinal muscular atrophy (SMA), knowing this information will be useful to doctors who want to provide the best counseling to their SMA patients considering motherhood. The study, “Pregnancy And Delivery In Women With Spinal…
The EU Joint Programme —Neurodegenerative Disease Research (JPND) is calling for proposals for pathway analysis of multinational research projects, focusing on neurodegenerative diseases such as spinal muscular atrophy (SMA). According to a JPND news piece, neurodegenerative disorders such as Alzheimer’s disease and other dementias and Parkinson’s disease are…
In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074). Biogen presented the data at the British Paediatric Neurology Association (BPNA)…
Patients with spinal muscular atrophy (SMA) are at risk of developing the metabolic disorder ketoacidosis, a less recognized complication that is easy to treat, according to a case study. Researchers behind the work, “Stress-Induced Ketoacidosis in Spinal Muscular Atrophy: An Under-Recognized Complication,” noted that testing for the condition, which…
Researchers may have discovered a new treatment for spinal muscular atrophy (SMA). Identifying a molecule that prevents the SMN2 gene from being read by protein-making machinery, they then showed that getting rid of it increased SMN protein levels. Importantly, the approach also enhanced the effectiveness of a splice-modification drug that works much like the…
A number of important discoveries, therapeutic developments, and events related to spinal muscular atrophy (SMA) were reported daily by SMA News Today throughout 2016. Now that the year is over, it is time to briefly review the articles that appealed most to our readers. Here are the 10 most-read articles of 2016,…
Patients with spinal and bulbar muscular atrophy (SBMA) may be at risk of developing respiratory complications due to a decline in the levels of genes responsible for muscle strength, according to a new study. The authors assert that monitoring the breathing decline in these patients may help provide better clinical…
On the eve of the weekend Christmas holiday, the U.S. Food and Drug Administration (FDA) approved Biogen’s therapy Spinraza (nusinersen) for the treatment of spinal muscular atrophy in both children and adults. The landmark approval marks the first time the federal agency has approved a therapy that directly treats the orphan disease, which is…
