Safety and efficacy of Zilganersen – Interventional – NCT04849741

Evaluating the safety and efficacy of Zilganersen, an antisense oligonucleotide targeting GFAP production

Clinicaltrial.gov Ionis website

AxD Outcomes – Observational – NCT02714764

Study on the natural history of Alexander disease

Clinicaltrial.gov Children’s Hospital of Philadelphia website