Research.Patient.info
Phosphomannomutase 2 Deficiency research hub
3 indexed studies · 1 currently recruiting
- active not recruitingPHASE2, PHASE3
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
This study is testing a new medicine called GLM101 for people with PMM2-CDG, a rare genetic condition. We want to see if it's safe and helps improve symptoms like balance and movement, comparing it to a dummy treatment.
United States · Belgium · Czechia - ongoing, recruitingTherapeutic exploratory (Phase II)
A Phase 2, Randomized, Open-Label, 24-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult, Adolescent and Pediatric Participants with PMM2-CDG
This study is testing a new medicine, GLM101, for people with PMM2-CDG. Researchers want to see if it's safe, how it affects the body, and if it helps with movement problems over 24 weeks.
Spain - enrolling by invitationPHASE2
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
This study offers continued access to a medicine called GLM101 for people with PMM2-CDG who have taken it before. The aim is to see how GLM101 affects them over a longer time, checking its safety and any lasting benefits. Participants will receive weekly infusions.
United States · Czechia · France