Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

计划入组 9 人 · 首次发布于 2009 年 9 月 14 日

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

计划入组 8 人 · 首次发布于 2001 年 11 月 1 日

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

计划入组 60 人 · 首次发布于 2005 年 6 月 6 日

Acid alpha-glucosidase gene therapy (Sarepta)

Recombinant human highly phosphorylated acid alpha-glucosidase

Recombinant adeno-associated virus acid alpha-glucosidase(University of Florida)

Alpha Metallurgical

United States

Zeno Alpha, Inc.

United States

Alpha Republic GmbH

Austria

Shionogi Initiates Phase 2 Trial for First Oral Substrate Reduction Therapy in Late-Onset Pompe Disease

AskBio Receives FDA Clearance to Begin Phase 1/2 Gene Therapy Trial for Late-Onset Pompe Disease

Advancements in Pompe Disease Treatment: A Pipeline Review