At the heart of rare disease treatments is research. Currently, 95% of all rare diseases still have no approved therapies. Although there has been an increased focus on rare diseases…
The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…
According to a story from Connecticut Children's, the hospital is one of the very first in the country to administer Zolgensma, a recently approved gene therapy for the rare genetic…
A Colorado mom is on a mission to raise money for her daughter's type 1 spinal muscular atrophy (SMA) treatment -- that would save her child’s life. Let's hear little Maisie…
SMARD is such a rare disease that only 100 children in the entire world have been diagnosed with it. SMARD stands for spinal muscular atrophy with respiratory distress. SMARD…
According to a story from The Washington Post, two families affected by the rare disease spinal muscular atrophy have cause for celebration after UnitedHealthcare agreed to cover their treatment with…
Meet Reece Anthony Rubino - a 13-year-old teen who loves video games, Disney World, and baseball. At just 3 months old, he was diagnosed with Type 1 spinal muscular atrophy…
For rare disease patients who face physical disabilities, even seemingly simple tasks can pose extreme difficulties. For instance, changing the channel on the television. Comcast Corp. previously created a voice…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
According to a story from BioSpace, on June 7th, 2019, Londyn Wright became the first baby in the US to receive Zolgensma, a recently approved gene therapy treatment for the…
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…
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