Okay, I’m going to be perfectly honest: I don’t quite know what to make of this. On the one hand, you have some excellent news for people living with mucopolysaccharidosis…
Hunter Syndrome, is a heartbreaking twist of genetic fate, and we celebrate that the FDA has granted Rare Pediatric Disease Designation to REGENXBIO's RGX-121 Gene Therapy. Hunter Syndrome causes seemingly…
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