Spinal muscular atrophy

Sheffield Children’s Hospital: Baby ‘doing OK’ after SMA drug first – BBC News

Markuss should be able to go home about two weeks after receiving…

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Evrysdi approved in Japan for the treatment of spinal muscular atrophy – Spinal News International

PTC Therapeutics has announced that Evrysdi (risdiplam) has been approved in Japan by the Japanese…

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Gene therapy gives families hope for babies with spinal muscular atrophy – UNSW Newsroom

A trial has revealed that babies with SMA treated with gene therapy are meeting the normal developmental milestones of any baby. [caption id=”attachment_1191860″ align=”aligncenter”…

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Because you’re worth it: Parents’ joy as Scots babies become first in UK to get £1.8m life-transforming, hope-giving treatment – The Sunday Post

Parents Margaret Paluszynska and Richard Winfield with baby Isabella at home in…

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Baby boy first to get £1.8m treatment on NHS for spinal muscular atrophy – ITV News

At just five months old Arthur Morgan from South London is offering hope to hundreds…

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Pioneering spinal treatment helping adults with debilitating condition – About Manchester

The first adult patient to be treated at Salford Royal with a new drug for…

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Baby boy is first to receive £1.8m treatment for spinal muscular atrophy on NHS – The Guardian

Arthur Morgan with his father in Evelina London children’s hospital where he…

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Spinraza: SMA patients win fight over treatment loophole – BBC News

Since 2019, Jake Ogborne has experienced a decrease in his mobility, and…

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My Daughter’s Rare Disease Has Given My Family a Community – Healthline

  When my husband, Jeremy, and I found out that…

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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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