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 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…
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 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…
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…
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 finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is caused by a mutation in the SMN1 gene. This mutation results in a loss of motor neurons causing weak and atrophied muscles.…
According to a publication from SMA News Today, Scotland is now the latest country to make spinal muscular atrophy (SMA) drug Spinraza available to patients at low or no cost…
Second Annual International Congress on Advanced Treatments in Rare Diseases The first International Congress on Advanced Treatments in Rare Diseases, otherwise known as RARE2018, was held in London, England. Now,…
A recent interview with David Alvarez and his mother in the CTPost explains how he manages to remain positive after being diagnosed with spinal muscular atrophy (SMA). David is a freshman…
According to a story from mirror.co.uk, a five-month-old girl named Maryam Malji was diagnosed with spinal muscular atrophy, a progressive, degenerative disease that is ultimately fatal without treatment. Her brother…
Spinal muscular atrophy (SMA) is a genetic condition which affects between 10,000 and 25,000 in the United States. While rare, it is currently the leading genetic cause of infant mortality. But,…
A recent article by NBC News is highlighting a growing concern among parents and professionals alike over the effectiveness of newborn genetic screening. There is a certain place between knowing…
According to a story from physiciansweekly.com, a recent report from the Institute for Clinical and Economic Review (ICER) suggests that, pending more data about overall price and longterm success rates,…
According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…
Who's in the mood to help grant a young girl her Christmas wish this year?! People who know 10-year-old Paola Vega describe her as a cute, spunky princess who can't…
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