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…
One of the wealthy regulars on the TV show Shark Tank made his millions by starting a home-based business selling tee shirts. An article in Bridgeport’s ctPost publication chronicles…
Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…
Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to…
As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…
According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…
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According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.…
Personalized Medicine You may have heard of personalized medicine. It's a new wave of healthcare that aims to provide individualized treatment to patients based on their own genetic makeup. The…
Spinal muscular atrophy (SMA) is a rare disorder, but sadly, it is still the most common genetic reason that children die before the age of 2. Not only that, but…
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