Alina Citovic was diagnosed with spinal muscular atrophy at age five, just three years ago. At the time of her diagnosis, Spinraza was already approved by the FDA. While the…
According to a story from SMA News Today, a recent case study has reported instances in two spinal muscular atrophy (SMA) patients in which repeated administrations of the therapy nusinersen…
Ever since he was a young boy, JR Trout, who lives with spinal muscular atrophy (SMA), had a sense that there was something different about him. For years, he never…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…
A report from the Neurological Alliance calls for improvements in treatment and care for the 150,000 rare neurological disease patients in the UK, including spinal muscular atrophy (SMA) patients. It…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy, or SMA, is a genetic disease that affects an individual’s central and peripheral nervous systems, as well as their…
Evrysdi, a treatment for spinal muscular atrophy (SMA), has started to make progress around the world in terms of approval. Brazil's regulatory agency, National Health Surveillance Agency, has approved the…
According to a story from SMA News Today, the Patient Access Network (PAN) Foundation announced that it is now offering financial assistance to help patients living with the rare disease…
As written in BioSpace, Genetech has recently presented new two-year data on their spinal muscular atrophy (SMA) treatment, evrysdi. The information was released at the 25th International Annual Congress of…
Recently, researchers sought to better understand the natural history and natural progression of type III spinal muscular atrophy (SMA). According to the AJMC, this study offers more insight into…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a genetic disease that affects the central and peripheral nervous systems, as well as voluntary muscle…
In recent years, gene therapy has expanded to enormous heights. This experimental technique allows scientists to add, delete, edit, or otherwise use genes to address genetic malfunctions and disorders. Now,…
Would you be shocked to find out that before Evrysdi, there were zero orally-administered drugs approved to treat patients with spinal muscular atrophy (SMA)? But now, things have changed. Last week,…
August is recognized as Spinal Muscular Atrophy Awareness Month. The goal of this event is to help spread information about this rare disorder among the general public and the broader…
By Lauren Taylor from In The Cloud Copy Biogen announced new data from the NURTURE trial of pre-symptomatic patients with spinal muscular atrophy (SMA). SMA is a rare, autosomal recessive…
Genentech has recently released the data from its SUNFISH trial, a study of the effects of risdiplam on spinal muscular atrophy (SMA) patients. Preliminary data from the JEWELFISH trial has…
Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…
by Jodee Redmond from In the Cloud Copy Emma Larson’s parents were not particularly concerned when their daughter wasn’t standing or walking by her first birthday. They figured that plenty of children had not reached…
According to a story from The Charlotte Weekly, local resident Kevin Tobin recently ran 400 laps around his house---the equivalent of a marathon---to help raise money for Dan Donoher, a…
As initially covered by MyLondon News, the family of a London boy with spinal muscular atrophy (SMA) is looking to advocate for others with this genetic disorder. Additionally, they…
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Zolgensma is a gene therapy developed by AveXis for the treatment of spinal muscular atrophy. It was approved by the FDA in May of 2019, and was later cleared for…
In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the Japanese Ministry of Health, Labour…
According to a story from Benzinga, the Novartis Company AveXis recently announced the results of long term studies testing the impact of Zolgensma, a gene therapy treatment that was approved…
The European Parliament has announced that they will be relaunching their "Network of Parliamentary Advocates for Rare Diseases." Its aim is to create a new policy framework which can improve…
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The gene therapy Zolgensma recently won approval last summer in 2019 to be used for infants who have spinal muscular atrophy. The company AveXis, the makers of the Zolgensma gene…
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