The U.S. Food and Drug Administration (FDA) has given fast track designation to Biohaven Pharmaceuticals‘ experimental medicine taldefgrobep alfa for the treatment of spinal muscular atrophy (SMA). The designation is granted to therapies that show potential in addressing serious conditions for which available treatments fall short. It is…
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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…
The Tufts Medical Center, a teaching hospital of the Tufts University School of Medicine, has been awarded a Muscular Dystrophy Association (MDA) grant to investigate patient access to treatments for spinal muscular atrophy (SMA) and other diseases. The project, led by James Chambers, PhD, will evaluate access to…
A gene-silencing therapy designed to lower the levels of two proteins was found to improve muscle strength and coordination in a mouse model of spinal and bulbar muscular atrophy (SBMA), a new study shows. The therapy, which targets the protein lysine-specific demethylase 1 (LSD1) and the protein arginine methyltransferase…
People with spinal muscular atrophy (SMA) types 2 and 3 who received Evrysdi (ridisplam) in the SUNFISH clinical trial and its extension part continued to see improvements or stabilizations in motor function after two years, according to published trial data. Notably, patients who were originally assigned to a…
Young children with spinal muscular atrophy (SMA) feel more fearful and sad, and may have a harder time recovering from distress than children who are healthy or have other conditions, a small study in India has found. These patients also seem to have more difficulty expressing their feelings or…
Most children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) retain motor function that exceeds what’s normally seen in untreated disease, according to a new analysis. In fact, a significant number of children who received the approved gene therapy were found to achieve one…
Some videos on YouTube, particularly in-depth ones made by healthcare professionals, provide high-quality and reliable information about treatments for spinal muscular atrophy (SMA), a new study highlights. But many of the examined videos concerning SMA treatments are neither high quality nor reliable, the researchers found, adding importance to identifying…
The motor function of children with spinal muscular atrophy (SMA) improves significantly in the early stages of treatment with Spinraza (nusinersen), namely within its two-month loading dose period, a study reported. No changes in nutritional status — as assessed by weight — were seen then, however, likely because…
In people with spinal muscular atrophy (SMA) who have been on long-term treatment with Spinraza (nusinersen), levels of the protein tau in the spinal fluid are decreased, a new study indicates. The findings imply that measuring levels of tau might be a useful marker for predicting the response…
