Trial Profile
An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.
Status:
Completed
Phase of Trial:
Phase I/II
Latest Information Update: 07 Nov 2021
Price :
$35
*
At a glance
- Drugs SAF 301 (Primary)
- Indications Mucopolysaccharidosis III
- Focus Adverse reactions
- Sponsors LYSOGENE
- 10 Feb 2014 New trial record