Weekly Spotlight - 07.11.24

Exploring caregivers' needs and advancements in SMA treatments and research.

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Spinal Muscular Atrophy UK: Christmas Jumper Day 2024

In the News

Caregivers Prioritise Safety in SMA Treatment Choices

Caregivers of children with spinal muscular atrophy (SMA) prioritise safety and breathing support over motor function improvements. A study highlights their preference for treatments with minimal risk and cost coverage. One-time intravenous infusions are favoured, with care preferred at local neurology departments. Understanding these priorities aids decision-making in treatment choices and policy development for SMA care.

Join Cure SMA's Year-End Campaign to Double Your Impact Today

Cure SMA's campaign, inspired by Hunter Rhodes, aims to raise £100,000 with a matching gift challenge. The Johns Family's £50,000 donation encourages community support for spinal muscular atrophy. Achievements include FDA-approved treatments and newborn screenings. Your support can empower those with SMA, fostering independence and enabling future breakthroughs for this condition.

New Mouse Model Paves Way for XL-SMA Gene Therapy Research

Professor Gillingwater's team has developed a mouse model for XL-SMA, a rare muscle-wasting condition. This model will help test UBA1-targeted therapies, offering hope for effective treatment. The research aims to understand XL-SMA's biology, paving the way for future therapies and potentially changing the future of muscle conditions.

Health Spotlight’s Spinal Muscular Atrophy is a Contentive publication in the Healthcare division