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

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 ...

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

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 ...

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] ...

The US Food and Drug Administration (FDA) has extended Regenxbio’s review timeline of the Biologics License Application (BLA) ...

RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome ...

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

REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) extended its review timeline of the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX ...

Denali Therapeutics posts narrower-than-expected Q2 loss. The company advances multiple rare disease therapies toward potential approvals.

Tividenofusp alfa BLA for Hunter syndrome accepted for priority review and assigned PDUFA target action date of January 5, 2026; company preparing for commercial launchDNL126 accelerated approval path ...