Survival outcomes in spinal muscular atrophy (SMA) are heavily influenced by age of onset, even among patients who are classified as having the same type of SMA, according to an analysis of more than three decades’ worth of data at a center in Thailand. In fact, data suggest…

Treatment with bicalutamide and trehalose extended survival and improved motor function in a mouse model of spinal and bulbar muscular atrophy (SBMA), a study has found. Both compounds had beneficial effects on some aspects of muscle fiber structure and reduced cell death. While bicalutamide also lowered levels of toxic…

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 introduction of Spinraza (nusinersen) as a treatment for spinal muscular atrophy (SMA) in Sweden was associated with trends of improved survival in children diagnosed with the disease before they were 2 years old, a nationwide population-based study shows. While these trends did not reach statistical significance relative…

Researchers in Hong Kong have shared a case report about how they were inspired and humbled by a teenage girl with spinal muscular atrophy (SMA) who got through a difficult hospital stay while maintaining a hopeful spirit and never losing sight of her goals. “Although the life of children…

Treatment with human fat tissue-derived mesenchymal stem cells (MSCs) may improve motor nerve cell health and survival in infants with spinal muscular atrophy (SMA) type 1, according to data from a small Phase 1 clinical trial in Iran. Notably, the stem cell treatment appeared to have effectively slowed disease progression…

Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Zolgensma safely and effectively halts disease progression in pre-symptomatic spinal muscular atrophy (SMA) infants, according to updated…

Genentech’s Evrysdi (risdiplam) improved survival in infants with spinal muscular atrophy (SMA) type 1, and allowed them to achieve developmental milestones not usually seen in the natural course of the disease, according to one-year data from the dose-finding part of the FIREFISH trial. While the trial is still ongoing…

Lowering the expression of the EphA4 gene — a gene that plays a major role in many neurological disorders — is not enough to improve motor function and survival in a mouse model of severe spinal muscular atrophy (SMA), a study has found. The study, “Lowering EphA4 Does…

Elevated levels of the protein alpha-COP,  involved in cellular transport and self-cleansing processes, extended survival in a mouse model of severe spinal muscular atrophy (SMA) without altering levels of the survival motor neuron (SMN) protein, according to a recent study. These results suggest this protein can modify SMA,…