Spinal muscular atrophy

Evrysdi improves, sustains motor function in spinal muscular atrophy – Healio

  Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained…

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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…

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Spinal muscular atrophy gene therapy coming to NHS – BioNews

  The NHS has agreed a deal to make…

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Parents celebrate new spinal muscular atrophy therapy – BBC News

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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The UK’s National Institute for Health and Care Excellence (NICE) has published draft guidance recommending…

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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:…

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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…

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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:…

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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…

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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…

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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…

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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…

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Canadian baby with spinal muscular atrophy to get ‘world’s most expensive drug’ – CTV News

  A little over…

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From no drugs to 3: Patients with spinal muscular atrophy now face hard choices – BioPharma Dive

  Jud Broadhurst was 14 years old when he…

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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…

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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…

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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…

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Thinking Outside the Box to Support My Son With Spinal Muscular Atrophy – Yahoo Lifestyle

  When a person…

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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…

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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…

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