Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…
Some promising news for the congenital adrenal hyperplasia (CAH) community! Spruce Biosciences is one step closer to a treatment for CAH after new phase 2a data showed the drug reduced…
According to a story from sectorpublishingintelligence.co.uk, studies utilizing data gathered in the real-world treatment setting are highlighting the characteristics of two different therapies for multiple sclerosis (MS) developed by the…
Every ultrasound Maggie had showed that her daughter, Aunika, was perfectly fine. The family was left in shock when she was born struggling to breathe. Baby Aunika was rushed to…
According to a story from Science Daily, the University of California San Francisco's Benioff Children's Hospitals have successfully treated an infant with juvenile myelomonocytic leukemia. The drug used for treatment…
We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…
In the US, medical costs come in the form of drug prescriptions, doctors visits, expensive surgeries, and visits to specialists. As originally reported in The Tyee, in Canada, patients are…
According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…
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