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Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…
Sometimes, rare diseases are so rare that they are just as unique as the individual who has it. One child was suffering from a fatal neurodegenerative disease, and the family…
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According to a recent article in SMA News Today, risdiplam (formerly RG7916) is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…
According to a story from finanznachrichten.de, the latest data from three phase 3 studies is giving further confirmation to the capability of the recently approved gene therapy Zolgensma to have…
According to a story from medicalxpress.com, Dr. Nathan Hoot is an emergency medical physician, but his role as a doctor doesn't entirely end when he leaves the hospital because his…
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A molecular biologist, Dr. Daria Julkowska, coordinates an international consortium in Paris that is largely funded through the 28-member European Union (EU). According to a report published in the Charcot-Marie-Tooth…
According to a story from biotech-now.org, the behavior of health insurance companies in the US is just one of the many problems that plagues our health system. While Americans pay…
Maisie Green Maisie Green is a 20-month-old little girl whose insurance has finally approved coverage for a life-altering spinal muscular atrophy treatment. Two weeks ago she received the singular infusion…
According to a story from Palo Alto Online, it was only a few months into the life of Aleksandr Kostanyan when parents Svetlana Sargsyan and Tigran Kostanyan realized that something…
According to a story from Mizzou News, Chris Lorson, who is the associate dean for Research and Graduate Studies at the school's College of Veterinary Medicine, recently earned four grants…
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…
The 2019 Annual SMA Conference Anaheim, California Cure SMA's annual conference is the largest conference in world focused on spinal muscular atrophy. The event includes and event for both patient…
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…
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