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…
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…
‘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…
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…
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…
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…
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…
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…
Spinal Muscular Atrophy awareness: Treatment advances offer hope to SMA patients – Firstpost
SMA is a rare inherited neuromuscular disorder that can can rob a…
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…
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…
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…