The brain has long been thought of as a singular organ, but a new study shows that the front and back parts of the brain actually develop completely independently of each other. This discovery may have key implications for research into diseases like spinal muscular atrophy (SMA), which affects…
News
A rapid, in-house genetic test allowed doctors in Japan to confirm a newborn’s diagnosis of spinal muscular atrophy (SMA) within hours and start treatment when the boy was 9 days old. The baby was readmitted to the hospital eight days after birth because of poor feeding and weight loss.
Children with spinal muscular atrophy (SMA) already living with symptoms experienced gains in motor abilities after receiving the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), a small Brazilian study found. The real-world report tracked the outcomes of seven youngsters in the South American nation who were treated with Zolgensma only…
Children and adults with spinal muscular atrophy (SMA), as well as their caregivers, may experience clinically relevant mental health symptoms, including anxiety and depression, a nationwide German study found. Among caregivers, those of patients with SMA type 1, the most common form of the disease, showed the greatest psychological…
Mindy Henderson has spent much of her life challenging the limits others have placed on her. When she was diagnosed with spinal muscular atrophy (SMA) — a genetic condition that causes muscles to weaken over time — as an infant, doctors told her parents that she might not live long…
Researchers at Cedars-Sinai are working to develop a potential stem cell treatment for three teenage siblings in California with an extremely rare form of spinal muscular atrophy (SMA) for which there are no available therapies targeting the disease’s underlying cause. The Vehling trio — sisters Emilie, 19, and Maggie…
More than a year after the U.S. Food and Drug Administration (FDA) rejected the muscle-strengthening agent apitegromab for spinal muscular atrophy (SMA) due to manufacturing issues, the agency has approved the add-on treatment for use by certain people with the rare genetic condition. The therapy’s developer…
A European survey of people living with spinal muscular atrophy (SMA) has found that while treatment options have expanded since the first medicine was approved in 2017, patient journeys remain far from straightforward. Data from the 2025 European Patient Experience Survey on SMA Medicines, Access, and Treatment Journeys…
A new blood test may offer a faster and simpler way to reliably diagnose most cases of spinal muscular atrophy (SMA), with results available in about one hour, a study showed. The LNA-PCR assay looks for homozygous SMN1 deletions, the most common SMA-causing mutation, in which a key section of…
Proteins involved in producing histones, which help package DNA inside cells, may take on new roles in mature nerve and muscle cells, a U.S.-Polish study found. The findings could provide new clues about spinal muscular atrophy (SMA). Researchers found that as nerve and muscle cells…
