As reported on drugs.com, REGENXBIO has disclosed that the U.S. Food and Drug Administration has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, both being developed for…
Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born. Newborn screening is a public health initiative that tests newborn babies for…
Newborn screening is an important public health initiative that checks for rare and serious genetic, blood, hormonal, or metabolic conditions at birth. The test collects blood through a heel…
617 words (source - 3% match) vs. 452 words (mine - 4% match) As our healthcare field continues to innovate and grow, we have seen more conversations regarding gene therapy…
Right now, there are only an estimated 10 people in Ireland who have been diagnosed with a form of mucopolysaccharidosis type I (MPS I) called Hurler syndrome. This is the…
Currently, the standards-of-care for patients with Hurler syndrome, or one of the forms of mucopolysacchardiosis type I (MPS I), include enzyme replacement therapy and hematopoietic stem cell transplants. However, Medical…
At the very end of March 2021, specialty pharmaceuticals company JCR Pharmaceuticals Co., Ltd. ("JCR") shared that its investigational drug candidate JR-171 received Orphan Drug designation from the European Medicines…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
The Malaysia Lysosomal Diseases Association (MLDA) was formed in 2011 by the hands of eight families. Their goal was to raise awareness and funds for those diagnosed with lysosomal storage…
The first patient has been treated in the Empowers study, a clinical trial investigating an experimental gene editing therapy for the treatment of mucopolysaccharidosis type I. The source article, which…
According to a story from PR Newswire, the biotechnology company REGENXBIO recently announced that the U.S. Food and Drug Administration (FDA) has granted its experimental gene therapy product RGX-111 Fast…
Haley Hayes is a normal pre-teen, yet she suffers from Pompe disease. Pompe disease is a genetic condition, which causes muscle and organ impairment. Early diagnosis and enzyme replacement therapy…
Say what you will about genetic testing—or testing in general—but North Carolina is finally feeling out the waters when it comes to newborn testing. The state is currently piloting a study…
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