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5. Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease. Peng J; Dalton J; Butt M; Tracy K; Kennedy D; Haroldsen P; Cahayag R; Zoog S; O'Neill CA; Tsuruda LS J Pharmacol Exp Ther; 2017 Feb; 360(2):313-323. PubMed ID: 27856936 [TBL] [Abstract][Full Text] [Related]
6. Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease. Yi H; Sun T; Armstrong D; Borneman S; Yang C; Austin S; Kishnani PS; Sun B J Mol Med (Berl); 2017 May; 95(5):513-521. PubMed ID: 28154884 [TBL] [Abstract][Full Text] [Related]
7. Efficient therapy for refractory Pompe disease by mannose 6-phosphate analogue grafting on acid α-glucosidase. Basile I; Da Silva A; El Cheikh K; Godefroy A; Daurat M; Harmois A; Perez M; Caillaud C; Charbonné HV; Pau B; Gary-Bobo M; Morère A; Garcia M; Maynadier M J Control Release; 2018 Jan; 269():15-23. PubMed ID: 29108866 [TBL] [Abstract][Full Text] [Related]
9. Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects. Colella P; Sellier P; Gomez MJ; Biferi MG; Tanniou G; Guerchet N; Cohen-Tannoudji M; Moya-Nilges M; van Wittenberghe L; Daniele N; Gjata B; Krijnse-Locker J; Collaud F; Simon-Sola M; Charles S; Cagin U; Mingozzi F EBioMedicine; 2020 Nov; 61():103052. PubMed ID: 33039711 [TBL] [Abstract][Full Text] [Related]
10. Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial. Klinge L; Straub V; Neudorf U; Schaper J; Bosbach T; Görlinger K; Wallot M; Richards S; Voit T Neuromuscul Disord; 2005 Jan; 15(1):24-31. PubMed ID: 15639117 [TBL] [Abstract][Full Text] [Related]
11. Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice. Ziegler RJ; Bercury SD; Fidler J; Zhao MA; Foley J; Taksir TV; Ryan S; Hodges BL; Scheule RK; Shihabuddin LS; Cheng SH Hum Gene Ther; 2008 Jun; 19(6):609-21. PubMed ID: 18500944 [TBL] [Abstract][Full Text] [Related]
12. [Adult form of Pompe disease]. Ziółkowska-Graca B; Kania A; Zwolińska G; Nizankowska-Mogilnicka E Pneumonol Alergol Pol; 2008; 76(5):396-9. PubMed ID: 19003770 [TBL] [Abstract][Full Text] [Related]
13. Design of Potent Mannose 6-Phosphate Analogues for the Functionalization of Lysosomal Enzymes To Improve the Treatment of Pompe Disease. El Cheikh K; Basile I; Da Silva A; Bernon C; Cérutti P; Salgues F; Perez M; Maynadier M; Gary-Bobo M; Caillaud C; Cérutti M; Garcia M; Morère A Angew Chem Int Ed Engl; 2016 Nov; 55(47):14774-14777. PubMed ID: 27774736 [TBL] [Abstract][Full Text] [Related]
14. Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease. Schaaf GJ; van Gestel TJM; In 't Groen SLM; de Jong B; Boomaars B; Tarallo A; Cardone M; Parenti G; van der Ploeg AT; Pijnappel WWMP Acta Neuropathol Commun; 2018 Nov; 6(1):119. PubMed ID: 30404653 [TBL] [Abstract][Full Text] [Related]
15. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk. Van den Hout JM; Kamphoven JH; Winkel LP; Arts WF; De Klerk JB; Loonen MC; Vulto AG; Cromme-Dijkhuis A; Weisglas-Kuperus N; Hop W; Van Hirtum H; Van Diggelen OP; Boer M; Kroos MA; Van Doorn PA; Van der Voort E; Sibbles B; Van Corven EJ; Brakenhoff JP; Van Hove J; Smeitink JA; de Jong G; Reuser AJ; Van der Ploeg AT Pediatrics; 2004 May; 113(5):e448-57. PubMed ID: 15121988 [TBL] [Abstract][Full Text] [Related]
16. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Kishnani PS; Corzo D; Nicolino M; Byrne B; Mandel H; Hwu WL; Leslie N; Levine J; Spencer C; McDonald M; Li J; Dumontier J; Halberthal M; Chien YH; Hopkin R; Vijayaraghavan S; Gruskin D; Bartholomew D; van der Ploeg A; Clancy JP; Parini R; Morin G; Beck M; De la Gastine GS; Jokic M; Thurberg B; Richards S; Bali D; Davison M; Worden MA; Chen YT; Wraith JE Neurology; 2007 Jan; 68(2):99-109. PubMed ID: 17151339 [TBL] [Abstract][Full Text] [Related]
17. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease. Byrne BJ; Geberhiwot T; Barshop BA; Barohn R; Hughes D; Bratkovic D; Desnuelle C; Laforet P; Mengel E; Roberts M; Haroldsen P; Reilley K; Jayaram K; Yang K; Walsh L; Orphanet J Rare Dis; 2017 Aug; 12(1):144. PubMed ID: 28838325 [TBL] [Abstract][Full Text] [Related]
18. Amifampridine tablets for the treatment of Lambert-Eaton myasthenic syndrome. Mantegazza R Expert Rev Clin Pharmacol; 2019 Nov; 12(11):1013-1018. PubMed ID: 31639317 [No Abstract] [Full Text] [Related]
19. Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers. Raben N; Fukuda T; Gilbert AL; de Jong D; Thurberg BL; Mattaliano RJ; Meikle P; Hopwood JJ; Nagashima K; Nagaraju K; Plotz PH Mol Ther; 2005 Jan; 11(1):48-56. PubMed ID: 15585405 [TBL] [Abstract][Full Text] [Related]
20. Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice. Zhu Y; Li X; McVie-Wylie A; Jiang C; Thurberg BL; Raben N; Mattaliano RJ; Cheng SH Biochem J; 2005 Aug; 389(Pt 3):619-28. PubMed ID: 15839836 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]