Woman With SMA on Forbes’ Top 30 List: ‘Work Ethic Outshines Disability’ – SMA News Today
From being a child actor to a…
Groups Join Forces to Push for SMA Newborn Screening in Scotland – SMA News Today
As part of ongoing efforts to have all newborns in the U.K. screened for spinal…
University of Oxford: First UK pilot study of newborn screening for spinal muscular atrophy launched in Oxford – India Education Diary
Oxford University is initiating a population-based newborn screening study in the Thames Valley. This study…
First UK pilot study of newborn screening for spinal muscular atrophy launched in Oxford – University of Oxford
In the UK, every five days a baby is born with spinal muscular atrophy (SMA)….
When Your Child Has Spinal Muscular Atrophy: Tools to Help You Cope – Healthline
Learning that your child has spinal muscular atrophy (SMA)…
Emirati girl with spinal muscular atrophy takes her first few steps at Abu Dhabi long-term care hospital – Gulf News
A therapist works with Ali, 6, and helps him communicate with visitors…
UK Expands Referral Criteria for Zolgensma to Heavier Children – SMA News Today
The U.K. has expanded the referral criteria for…
£1.8m drug gives Cheshire baby a second chance at life after terminal diagnosis – Cheshire Live
A drug that cost £1.8m has…
‘Miracle’ Cystic Fibrosis Treatment For Children on The NHS – NHS England
Hundreds of children are set to benefit from a ‘miracle’ treatment for cystic fibrosis, which…
Top 5 Most-Read Spinal Muscular Atrophy Stories of 2021 – AJMC.com Managed Markets Network
The top 5 most-read spinal muscular atrophy stories of 2021 on AJMC.com covered a variety…
How does the NHS decide who they pay to treat – and who misses out? — Daily Mail
The numbers are mind-blowing. In England alone, the NHS spends an average of £52 million…
As trials lag on Risdiplam’s effectiveness in adults, the drug remains out of reach for many – ABC News
Eleanor, with her sister Kirsten (left) and mother Karen, wants Risdiplam to…
NICE recommends risdiplam for spinal muscular atrophy – European Pharmaceutical Review
The National Institute for Health and Care Excellence (NICE) has published new draft guidance recommending…
Gene Therapy Improves Muscle Function for Children With Spinal Muscular Atrophy – Technology Networks
Children with spinal muscular atrophy (SMA) carry specific gene defects which cause progressive muscle wasting…
Ideal Therapy Aids Motor Skills and Breathing, Taken Orally: UK Survey – SMA News Today
Motor function and stabilizing or improving breathing — along with oral administration — are the…
Significant boost for children with Spinal Muscular Atrophy as new gene therapy is approved for use in Ireland – Irish Medical Times
Children’s Health Ireland (CHI), St James’s Hospital, and…
Babies with SMA able to swallow with Evrysdi® treatment – European Pharmaceutical Review
Roche have announced positive data from its ongoing open label study, demonstrating the efficacy and…
‘It really is a miracle drug’: Yorkshire boy with spinal muscular atrophy receives ‘most expensive drug in the world’ at Sheffield Children’s Hospital – The Yorkshire Post
Rosie-Mae Walton and Wes Powell with their son Marley as he recovers…