Spinal muscular atrophy

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…

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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…

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‘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…

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Spinal Muscular Atrophy: Why An Early Diagnosis And Treatment Is Crucial For Patients – ABP Live

  Over the years, the unmet needs of patients with Spinal…

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New SMA Tracker Reveals Gaps in Access for People Living with SMA in Europe – EURACTIV PR

Biogen Inc. today announced the launch of the Spinal Muscular Atrophy (SMA) Policy and Access…

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Risdiplam improves motor function in infants with spinal muscular atrophy – Medical Xpress

Research on SMA goes back more than 50 years. Credit: Children’s Hospital…

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Moray tot with Spinal Muscular Atrophy is superhero – The Northern Scot

THE family of a two-year-old boy diagnosed with an incurable condition one week before the first coronavirus lockdown have thanked family, friends and…

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Little boy with Spinal Muscular Atrophy is a real superhero – Inverness Courier

THE family of a two-year-old boy diagnosed with an incurable condition one week before the first coronavirus lockdown have thanked family, friends and…

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Spinal Muscular Atrophy awareness: Treatment advances offer hope to SMA patients – Firstpost

SMA is a rare inherited neuromuscular disorder that can can rob a…

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7-year-old Bronx boy with Spinal Muscular Atrophy sworn in as NYPD officer for a day – WABC-TV

A 7-year-old boy from the Bronx had his dream come true Monday, as the NYPD…

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The ‘most expensive drug in the world’, how it works and the devastating disease it treats – The Conversation UK

Spinal muscula atrophy causes muscle wasting and loss of control, usually starting…

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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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