ClinicalTrials.gov
public full text
Public Domain
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to ERT with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until the conclusion of negotiations of avalglucosidase alfa in France under the standard reimbursement framework on each indication independently, infantile (IOPD) and late (LOPD), or until June 2027, whichever comes first. \- Study visit frequency: every 2 weeks
Conceptio Open Archive
Analyze & explore related research in Conceptio
Connect this record directly into your research workflow or AI agent. Explore citations across open repositories, query full text, and export structured proof bundles.
This document is indexed with metadata only: full text is not available in the archive for this record. Open the official source ↗
Related documents
- High Dose or High Dose Frequency Study of Alglucosidase Alfa#1126395
- Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug#869220
- Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease#1172292
- A Comparison of the Efficacy of Amisulpride and Placebo in the Prevention of PONV in Patients at Moderate-to-high Risk of PONV.#904380
- AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease#1111895
Record · ID 849800
Retrieved via Conceptio: every document is proof-bundled with source, license, and retrieval metadata.
Retrieved via Conceptio: every document is proof-bundled with source, license, and retrieval metadata.
Bring this data into your agent: Connect via MCP or API · 20 free credits to start · no card required
Read the Docs → · View CLI source on GitHub · Conceptio Open Knowledge Archive
Read the Docs → · View CLI source on GitHub · Conceptio Open Knowledge Archive