Evrysdi improves, sustains motor function in spinal muscular atrophy – Healio
Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained…
Genentech : Data On Evrysdi Shows Improvement Of Motor Function In Spinal Muscular Atrophy Patients – Nasdaq
Genentech’s, a member of the Roche Group (RHHBY), Evrysdi or risdiplam continues to demonstrate improvement…
Spinal muscular atrophy gene therapy coming to NHS – BioNews
The NHS has agreed a deal to make…
NICE approves gene therapy for spinal muscular atrophy – European Pharmaceutical Review
The UK’s National Institute for Health and Care Excellence (NICE) has published draft guidance recommending…
Spinal muscular atrophy: NHS to use world’s most expensive drug to treat babies with rare genetic disorder – iNews
NHS England said the expensive drug has proved effective against SMA (Photo:…
Novartis and NHS England reach ‘landmark’ deal for Zolgensma gene therapy for SMA – PMLiVE
Novartis Gene Therapies and NHS England have reached a ‘landmark’ deal for Zolgensma gene therapy…
NHS to use world’s most expensive drug to treat spinal muscular atrophy – The Guardian
The NHS says it has acquired Zolgensma at a significant discount. Photograph:…
Spinal Muscular Atrophy Cure: How Close Are We? – Healthline
Spinal muscular atrophy (SMA) is a rare genetic condition that impairs a person’s ability to…
The family desperate for a miracle drug that could help their son walk and talk – Manchester Evening News
Haris’ family hope the NHS will fund zolgensma – a one-off gene…
Top 5 Most-Read SMA Stories of 2020 – AJMC.com Managed Markets Network
The top 5 most-read stories about spinal muscular atrophy (SMA) of 2020 on AJMC.com focused…
Eight-week-old baby with spinal muscular atrophy could be saved by a single jab – Daily Mail
Like a ‘punch in the stomach’, is how Megan Willis felt on hearing that her…
From no drugs to 3: Patients with spinal muscular atrophy now face hard choices – BioPharma Dive
Jud Broadhurst was 14 years old when he…
Study Findings Offer More Understanding of Natural History of Type III SMA – AJMC
By assessing longitudinal changes in functional status, researchers are offering a better understanding of the…
How advances are changing the standard of care for patients with spinal muscular atrophy – Pharmafield
This August is Spinal Muscular Atrophy (SMA) Awareness Month. It’s the perfect time to celebrate…
Treatment Is Key for All With SMA, But Gains Will Differ – SMA News Today
Treating newborns whose spinal muscular atrophy (SMA) is discovered in screening programs — before symptoms…
How different generations living with SMA can teach one another – SMA News Today
Paying it forward Meet Regina and Al. Their friendship and respect for one another all started…
Coronavirus: The shielders turning the word ‘vulnerable’ on its head – BBC News
This weekend shielding will officially stop. The ‘elderly and vulnerable’ who have followed stringent rules will re-join society and follow the same social…