Researchers have developed a two-dimensional (2D) model that mimics the crosstalk between nerve and muscle cells. They intend to use it to screen large numbers of compounds in the hunt for treatments for spinal muscular atrophy (SMA) and other neuromuscular diseases. “We want to start by seeing if we…
Being on Spinraza (nusinersen) for up to three years results in a gain in motor function for children with spinal muscular atrophy (SMA) type 2, with the youngest children benefiting the most from treatment, a small study has found. “It is therefore important to start the treatment as…
Children with spinal muscular atrophy (SMA) type 1 may be at an increased risk of developing kidney stones, suggesting the condition may be more common than previously thought, according to a small study in Turkey. It’s possible that nephrolithiasis — the medical term for kidney stones — had been…
How spinal muscular atrophy (SMA) manifests among people who carry four copies of the SMN2 gene varies, ranging from type 2 to type 4, with functional abilities showing a tendency to decline with age, a study in Italy finds. Understanding how the disease progresses over time and what influences…
A first patient has been dosed in a Phase 2 clinical trial evaluating the safety and efficacy of NMD670, an oral treatment candidate for spinal muscular atrophy (SMA) type 3, its developer, NMD Pharma, announced. Called SYNAPSE-SMA (NCT05794139), the trial is now recruiting adults, ages 18 to…
Scholar Rock announced that a Phase 3 clinical trial of its experimental muscle-targeting therapy apitegromab has finished enrolling children and young adults with spinal muscular atrophy (SMA) types 2 or 3, who are unable to walk but can sit independently. “Completing SAPPHIRE enrollment is a critical…
The Muscular Dystrophy Association (MDA) has awarded one of 37 new research grants to a professor studying the role of survival motor neuron or SMN — the protein that’s missing in people with spinal muscular atrophy (SMA) — in disease development and treatment. Winning the three-year grant — in…
Biohaven Pharmaceuticals has completed the enrollment of children and young adults with spinal muscular atrophy (SMA) in a pivotal Phase 3 trial in the U.S. and Europe that is testing its experimental muscle-targeting therapy taldefgrobep alfa. The company had expected to recruit about 180 patients of all…
In an African American toddler, neuromuscular symptoms of a severe form of nutritional rickets — a condition characterized by weak or soft bones due to dietary deficiencies — were initially mistaken as spinal muscular atrophy (SMA) type 3, according to a new case study. Researchers say the overlapping symptoms…
The European Commission (EC) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old, making the therapy now available to treat SMA patients across all age ranges in Europe. Evrysdi was first approved in the European Union…
