The 'Omirge Sen' Foundation launched a video campaign for Rare Disease Day 2026 to raise awareness about hereditary neuromuscular diseases, including Duchenne muscular dystrophy and spinal muscular atrophy. This initiative highlights the stories of affected families and aims to support those impacted by these conditions.

The 'Omirge Sen' Foundation launched a video for Rare Disease Day 2026 to raise awareness about hereditary neuromuscular diseases, including Duchenne muscular dystrophy and spinal muscular atrophy. This initiative highlights the stories of families affected by these conditions.

The DySMA trial investigates swallowing development in infants and toddlers with spinal muscular atrophy compared to healthy controls. This prospective study provides insights into the impact of therapy on swallowing abilities in affected children.

A recent study published in PubMed details the clinical diagnosis and genetic analysis of a rare case involving both Duchenne muscular dystrophy and spinal muscular atrophy. This research contributes to the understanding of these conditions and may inform future diagnostic and therapeutic approaches.

Research reveals that organ-specific redox imbalances in spinal muscular atrophy (SMA) mice can be partially corrected using SMN antisense oligonucleotides. This study contributes to understanding the biochemical pathways involved in SMA and potential therapeutic approaches.

FDA approves Itvisma (onasemnogene abeparvovec-brve) for spinal muscular atrophy, targeting patients aged 2 and older with confirmed SMN1 gene mutations. This gene therapy represents a significant advancement in treatment options for SMA.