I am/have/had
I am looking for
Advanced Filters
Found 17 clinical trials
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
This clinical study aims to investigate the long-term safety and durability of GLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.
gene therapy
fabry's disease
deficiency
glycosphingolipids
breakdown
- 0 views
- 16 Feb, 2024
- 1 location
An Open-Label Study to Investigate the Safety of Single Ascending Doses in Children and Adolescents With Dravet Syndrome
Stoke Therapeutics is evaluating the safety and tolerability of single ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency and quality of life will be measured as secondary endpoints in this open-label study.
seizure
scn1a gene
tonic
fever
dravet syndrome
- 0 views
- 05 Aug, 2020
- 1
- 2