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 enzyme delivery to the brain and body, normalizing key …
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.
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy …
Jan 9, 2025 · Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with...
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 1/2 study results showed normalization of cerebrospinal fluid heparan sulfate and improved lysosomal function biomarkers.
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 Area hospital, there...
Denali receives FDA breakthrough status for Hunter syndrome drug
Jan 8, 2025 · Denali also plans to file its lead drug, DNL310 for Hunter Syndrome, for Accelerated Approval, with potential approval by the end of 2025. Positive topline data for another orphan disease drug,...
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 MPS Society provides frequent updates on news and events related to …
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.
New Interim Data Reported on DNL310 for Hunter Syndrome …
Sep 26, 2023 · Additional biomarker findings from up to 2 years of DNL310 treatment suggest positive changes in adaptive behavior and cognition as well as improvement in auditory function among pediatric patients with Hunter syndrome.
Project Alive | A Cure Within Reach
Hunter syndrome is a rare genetic disease that is progressively debilitating, life-limiting and without a cure. It occurs almost exclusively in males and currently affects approximately 500 boys in the U.S. and less than 2,000 worldwide.