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

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 ...

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

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] ...

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 ...

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 ...

Regenxbio (RGNX) announced that the U.S. Food and Drug Administration extended its review timeline of the Biologics License Application for ...