The investigational treatment risdiplam (RG7916) improved motor function in patients with type 1, 2 or 3 spinal muscular atrophy (SMA), according to preliminary results from the first part of two ... Read more
Walking ability in type 3 SMA patients is significantly impaired during adolescence, according to recent research.
Type 3 SMA, also known as Kugelberg-Welander disease, is a milder form of SMA. ... Read more
Administering stem cell therapy to the fetuses of mice with spinal muscular atrophy (SMA) type 3 improved the animals’ movement and survival after birth, a study reports. The research, “Prenatal transplantation of ... Read more
Spinal muscular atrophy (SMA) is a genetic disease that affects mostly children, but comes in different forms. The severe neuromuscular disease affects a person’s ability to participate in daily life ... Read more
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