The FDA has postponed its decision date for Regenxbio’s Hunter syndrome gene therapy to review additional longer-term ...

The extension was needed to review newly submitted long-term clinical data for all patients in the pivotal study following an FDA information request.

GC Biopharma, a leading global pharmaceutical company based in South Korea, announced today that it has revealed the delivery mechanism of Hunterase (idursulfase beta), a recombinant enzyme ...

Hunter syndrome is what's known as an "orphan disease" — it's so rare that not much research is devoted to it. This orphan disease got a mother.

Delays from the Food and Drug Administration continue to pile up, with Maryland-based Regenxbio the latest to report the review of one of its assets ...

US FDA extends review timeline of Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II: Rockville, Maryland Wednesday, August 20, 2025, 18:00 Hrs [IST] ...

From the San Bernardino County mountain community of Crestline, a mother is waging a war against rare diseases. Kristin McKay ...

India has over 180 children with Hunter Syndrome, but many people are not even aware of this genetic disorder which mostly ...