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

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 ...

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’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).

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] ...

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 ...

Hunter Syndrome is an extremely rare disease. There are only about 2,000 known cases around the world and one is right here in the metro.

Hunter syndrome will eventually cause neurological, cardiac and respiratory problems. In some children, it mimics an autism spectrum disorder. Ethan has some cognitive and behavioral issues.

Hunter’s syndrome is a lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (I2S). The disease was first described in 1917 by physician Charles A. Hunter (1873 ...

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

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