Ultragenyx
FDA Declines Approval of Ultragenyx’s Sanfilippo Gene Therapy Due to Manufacturing Issues
FDA; Ultragenyx; Sanfilippo syndrome type A; gene therapy; manufacturing issues; UX111; regulatory setback; complete response letter; approval delay
Ionis Advances to Phase 3 Trial for Angelman Syndrome Treatment ION582
Ionis Pharmaceuticals, Angelman syndrome, ION582, Phase 3 trial, antisense oligonucleotides, neurological conditions, rare diseases, Biogen, Ultragenyx Pharmaceutical
Ultragenyx Advances Gene Therapy for Glycogen Storage Disease Type Ia Following Phase 3 Success
Ultragenyx, gene therapy, glycogen storage disease type Ia, DTX401, Phase 3 study, GlucoGene, rare genetic diseases
Ultragenyx’s GTX-102 Shows Promising Results in Mid-Stage Angelman Syndrome Study, Pivotal Study on the Horizon
Ultragenyx, GTX-102, Angelman Syndrome, Mid-Stage Study, Pivotal Study, Antisense Oligonucleotide, Cognitive Improvement, Behavioral Improvement, Sleep Improvement, Gross Motor Improvement