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…
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…
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”…
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…
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…
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…
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…
Spinraza: SMA patients win fight over treatment loophole – BBC News
Since 2019, Jake Ogborne has experienced a decrease in his mobility, and…
My Daughter’s Rare Disease Has Given My Family a Community – Healthline
When my husband, Jeremy, and I found out that…
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The UK’s National Institute for Health and Care Excellence (NICE) has published draft guidance recommending…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…