Furthermore, AJ201-treated subjects showed improvements in the physical function component ... "AnnJi reports outcomes from trial of spinal bulbar muscular atrophy therapy" was originally created ...

We can improve the quality of life for children affected by SMA and similar disorders, ensuring they receive the multidisciplinary support and care needed to thrive and lead fulfilled lives.

Back to Healio An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular atrophy showed sustained motor function improvements at 48 months ...

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest ...

More information: Richard S. Finkel et al, Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy, New England Journal of ...

05. Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of ...

Spinal muscular atrophy is a genetically inherited disorder ... At your appointment, the doctor will perform a physical exam and request important medical information, like any family history ...

Zolgensma (onasemnogene abeparvovec) is the only approved gene therapy for spinal muscular atrophy. The therapy replaces the function of the missing or non-working SMN1 gene to halt disease ...

Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the ...

in infants with spinal muscular atrophy (SMA). The study's results add further support for gene therapy as a treatment modality that can deliver durable transformative effects for these vulnerable ...