FDA Grants Breakthrough Therapy Designation to Denali’s …
Jan 9, 2025 · Tividenofusp alfa received FDA breakthrough therapy designation for Hunter syndrome, with a biologics license application planned for early 2025. The therapy optimizes …
FDA Grants Breakthrough Therapy Designation to Tividenofusp …
Jan 9, 2025 · Tividenofusp alfa received FDA breakthrough therapy designation for Hunter syndrome, enabling expedited review due to promising preliminary clinical evidence. Phase …
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy …
Jan 9, 2025 · DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter …
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy
Jan 9, 2025 · Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical, …
Hunter Syndrome | Patient Resources | Medical News
Staying informed on medical progress can give you insights into the latest advances in managing Hunter syndrome. Below are some important resources to keep you up to date. The National …
Denali’s DNL310 Eyes Accelerated FDA Approval With
Sep 4, 2024 · Denali Therapeutics plans to seek accelerated FDA approval for DNL310 in Hunter syndrome, showing significant biomarker improvements and clinical benefits in ongoing trials.
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy …
Jan 8, 2025 · Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical, …
North Texas teen offers hope to others with Hunter Syndrome
Jan 16, 2025 · Fifteen-year-old Dominic Henriquez from Prosper, Texas, is bringing hope to boys like him who live with Hunter syndrome, a rare genetic disorder that primarily affects males.
Fetal therapy offers hope for unborn patients suffering from ... - CBS News
Jan 24, 2024 · Some parents-to-be have to face the terrifying news that their unborn child has been diagnosed with a life-threatening genetic disorder. But thanks to a medical team at a Bay …
Groundbreaking gene therapy trial for Hunter syndrome opens
The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.