Duke Medical Center
Durham, United States
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Pompe disease
FollowWhat is new in Pompe disease research, which clinical trials are open, and who works on it. From public registries and PubMed, with the source and date of every fact.
Cure status
- avalglucosidase alfaPhase 3 · NCT04910776
- Cipaglucosidase alfaPhase 3 · NCT04808505 · NCT03911505
- MiglustatPhase 3 · NCT04808505 · NCT03911505
- Alglucosidase alfa (GZ419829)Phase 2 · NCT03019406
- Avalglucosidase alfa (GZ402666)Phase 2 · NCT03019406
- ERTPhase 2 · NCT07750990
- S-606001Phase 2 · NCT07750990 · NCT07787650 · NCT07123155
- AB-1009 (GAA Gene)Phase 1/2 · NCT07282847
- GC301Phase 1/2 · NCT06391736 · NCT05793307
- SPK-3006Phase 1/2 · NCT04093349
- zocaglusagene nuzaparvovecPhase 1/2 · NCT04174105
- Aldurazyme (laronidase)Phase 1 · NCT04532047
- DNL952Phase 1 · NCT07354724
- CRG003 injectionEarly phase 1 · NCT06178432
Closest to patients
- Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaClinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaSeattle Children's Hospital- Site Number : 8400003 · Seattle, United States + 15 more hospitalsPhase 3Results expected 2026
- ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDGunma University Hospital · Gunma, Japan + 17 more hospitalsPhase 3Results expected 2027
- A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18UPMC Hospital of Pittsburgh · Pittsburgh, United States + 17 more hospitalsPhase 3Results expected 2027
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Latest news
Results, new trials and publications, newest first. Every item links to its source.
Results and new studies, newest first, in plain words.
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Case report
Statin-disclosed acid maltase deficiency.
Statin-disclosed acid maltase deficiency.
Journal of internal medicine
PubMed · PMID 16018797Publication
[Assessment of cardiac sympathetic nerve activity in children with chronic heart failure using quantitative iodine-123 metaiodobenzylguanidine imaging].
[Assessment of cardiac sympathetic nerve activity in children with chronic heart failure using quantitative iodine-123 metaiodobenzylguanidine imaging].
Journal of cardiology
PubMed · PMID 11190582Case report
Renal magnesium wasting, hypomagnesemic hypocalcemia, hypocalciuria and osteopenia in a patient with glycogenosis type II.
Renal magnesium wasting, hypomagnesemic hypocalcemia, hypocalciuria and osteopenia in a patient with glycogenosis type II.
American journal of nephrology
PubMed · PMID 11093001
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Groups with active Pompe disease work.
Duke University
Durham, United States
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Erasmus MC
Rotterdam, Netherlands
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Ludwig-Maximilians-Universität München
Munich, Germany
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University of Florida
Gainesville, United States
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Amicus Therapeutics (United States)
Cranbury, United States
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Newcastle University
Newcastle upon Tyne, United Kingdom
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University of California, Irvine
Irvine, United States
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LMU Klinikum
Munich, Germany
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Salford Royal NHS Foundation Trust
Salford, United Kingdom
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Amicus Therapeutics (United Kingdom)
Gerrards Cross, United Kingdom
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University of Messina
Messina, Italy
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