According to a story from Australasian Science Magazine, the dawn of the gene therapy "revolution" is upon us. While there are only a handful of gene therapies that have actually…
According to a story from PR Newswire, the Novartis Company AveXis has issued an announcement detailing special access programs that will help patients access Zolgensma, the first ever single use…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
According to a story from PR Newswire, the Novartis company AveXis recently announced that the US Food and Drug Administration (FDA) has approved Zolgensma, making it the first ever gene…
According to a story from thejournal.ie, Ireland's Health Services Executive (HSE) is facing renewed pressure from activists and patient advocates for a reasonable coverage decision regarding Spinraza, which is currently…
Selon un article de BBC News, le père Shakil Malji demande depuis des mois que sa jeune fille Maryam, atteinte d’une maladie rare et mortelle appelé l’atrophie musculaire spinale, puisse…
According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…
According to a story from The Jerusalem Post, Adrian Krainer is a prominent molecular geneticist and biochemist. His research was also pivotal in developing the first-ever disease modifying treatment for…
According to a story from BNN Bloomberg, data from three studies suggest that Zolgensma, a gene therapy for spinal muscular atrophy developed by Novartis, could be useful to all patients…
In February, Li Kegiang, Premier of the State Council, announced that the Chinese government was going to take steps to improve rare disease patients access to medicine. As part of…
According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…
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In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…
According to a story from globenewswire.com, the biotechnology company Biogen recently released data from an open label study that was testing the company's spinal muscular atrophy drug nusinersen (marketed as…
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According to an article in Biospace, the FDA is currently reviewing data from the Phase III STRIVE clinical trial for possible approval of Zolgensma as gene therapy for spinal…
According to a press release from the American Academy of Neurology published by EurekAlert, new research published in Neurology further suggests that established spinal muscular atrophy (SMA) drug nusinersen could…
Anna Landre is a sophomore at Georgetown University in Washington, D.C. It’s a good school, you’ve probably heard of it. On top of the normal stresses of her college life,…
According to a story from inpublic.globenewswire.com, the Novartis company AveXis has released interim data from an ongoing Phase 3 clinical trial of Zolgensma, a gene therapy drug that is being…
According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…
According to a story from Radio NZ, two grandmothers have taken a central role in getting a petition delivered to the parliament of New Zealand. This petition is calling for…
According to a story from BNN Bloomberg, gene therapies are without a doubt an upcoming advancement in care and treatment for many diseases that have a genetic origin. This includes…
Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…
According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
Avraham Steinberg recently spoke at RARE2019, the 2nd International Congress on Advanced Treatments in Rare Diseases. It was held on March 4th and 5th in Vienna, Austria. His talk focused…
According to a story from Xtalks, there has been an uptick in research related to rare diseases and the development of new therapies for them. Despite this, only a small…
Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…
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