According to a recent article, the family of a young boy from Australia who is diagnosed with the rare genetic disease mucopolysacchariodosis type 1 (MPS 1) is sharing his story to…
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…
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 a story from governmenteuropa.eu, Professor Susanne Kircher, who is associated with the Austrian MPS Society and the Medical University of Vienna, writes about the state of rare diseases…
According to an article from R&D, researchers at the New York City-based Icahn School of Medicine at Mount Sinai have entered an agreement with two pharmaceutical companies to aid in…
The United States Food and Drug Administration has awarded Rare Pediatric Disease Designation to a potential drug for Mucopolysaccharidosis type I. For more detailed information about this news, you can…
According to a story from PR Newswire, a new program called The Lantern Project is working to provide a free testing program that allow doctors to more quickly diagnose patients…
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…
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