LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS

计划入组 477 人 · 首次发布于 2026 年 7 月 6 日

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

计划入组 14 人 · 首次发布于 2015 年 2 月 3 日

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

计划入组 5 人 · 首次发布于 2017 年 8 月 21 日

Cipaglucosidase alfa(Pombiliti)

Avalglucosidase alfa(Nexviazyme, Nexviadyme)

Pompe disease(Codexis)

Rare Disease Research, LLC

CASTLEMAN DISEASE COLLABORATIVE NETWORK

United States

CURE RARE DISEASE, INC.

United States

Amicus Therapeutics Secures Japan Approval for Pombiliti + Opfolda Combination Therapy for Late-Onset Pompe Disease

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