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

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

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 ...

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] ...

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.

RegenXBio ( ($RGNX) ) has issued an announcement. On August 18, 2025, REGENXBIO announced that the FDA has extended the review timeline for its ...

Explore the competitive landscape of Mucopolysaccharidosis II (Hunter Syndrome) therapeutics with our detailed report. India will lead in diagnosed cases by 2024 and 2029. The market currently offers ...