Trial Profile
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Status:
Active, no longer recruiting
Phase of Trial:
Phase II/III
Latest Information Update: 21 Feb 2024
Price :
$35
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At a glance
- Drugs ABO 102 (Primary)
- Indications Mucopolysaccharidosis III
- Focus Registrational; Therapeutic Use
- Acronyms Transpher A
- Sponsors Ultragenyx Pharmaceutical
- 15 Feb 2024 According to an Ultragenyx Pharmaceutical media release, based on data from this trial, discussions are ongoing with the FDA seeking an accelerated review path for UX111.
- 15 Feb 2024 According to an Ultragenyx Pharmaceutical media release, results from this trial were presented at the 20th Annual WORLDSymposium™ earlier this month.
- 06 Feb 2024 Results ( data cut-off ,August 2023, n=17) presented in the Ultragenyx Pharmaceutical Media Release