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 ...

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).

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 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’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 ...

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 ...

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

Hunter syndrome results from the absence of an enzyme needed to break down cellular waste. Without it, waste builds up in the body, causing progressive damage to various systems.