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Items: 1 to 20 of 149

1.

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease.

Papadopoulos C, Orlikowski D, Prigent H, Lacour A, Tard C, Furby A, Praline J, Solé G, Hogrel JY, De Antonio M, Semplicini C, Deibener-Kaminsky J, Kaminsky P, Eymard B, Taouagh N, Perniconi B, Hamroun D, Laforêt P; French Pompe Study Group.

Mol Genet Metab. 2017 Sep;122(1-2):80-85. doi: 10.1016/j.ymgme.2017.06.007. Epub 2017 Jun 20.

PMID:
28648663
2.

Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.

Hundsberger T, Rösler KM, Findling O.

J Neurol. 2014 Sep;261(9):1684-90. doi: 10.1007/s00415-014-7402-z. Epub 2014 Jun 13.

PMID:
24923245
3.

Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.

Case LE, Bjartmar C, Morgan C, Casey R, Charrow J, Clancy JP, Dasouki M, DeArmey S, Nedd K, Nevins M, Peters H, Phillips D, Spigelman Z, Tifft C, Kishnani PS.

Neuromuscul Disord. 2015 Apr;25(4):321-32. doi: 10.1016/j.nmd.2014.12.004. Epub 2014 Dec 19.

4.

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.

Strothotte S, Strigl-Pill N, Grunert B, Kornblum C, Eger K, Wessig C, Deschauer M, Breunig F, Glocker FX, Vielhaber S, Brejova A, Hilz M, Reiners K, Müller-Felber W, Mengel E, Spranger M, Schoser B.

J Neurol. 2010 Jan;257(1):91-7. doi: 10.1007/s00415-009-5275-3. Epub 2009 Aug 1.

PMID:
19649685
5.

Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.

van der Ploeg A, Carlier PG, Carlier RY, Kissel JT, Schoser B, Wenninger S, Pestronk A, Barohn RJ, Dimachkie MM, Goker-Alpan O, Mozaffar T, Pena LD, Simmons Z, Straub V, Guglieri M, Young P, Boentert M, Baudin PY, Wens S, Shafi R, Bjartmar C, Thurberg BL.

Mol Genet Metab. 2016 Sep;119(1-2):115-23. doi: 10.1016/j.ymgme.2016.05.013. Epub 2016 May 19.

6.

Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.

Angelini C, Semplicini C, Ravaglia S, Bembi B, Servidei S, Pegoraro E, Moggio M, Filosto M, Sette E, Crescimanno G, Tonin P, Parini R, Morandi L, Marrosu G, Greco G, Musumeci O, Di Iorio G, Siciliano G, Donati MA, Carubbi F, Ermani M, Mongini T, Toscano A; Italian GSDII Group.

J Neurol. 2012 May;259(5):952-8. doi: 10.1007/s00415-011-6293-5. Epub 2011 Nov 12.

PMID:
22081099
7.

36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy.

Regnery C, Kornblum C, Hanisch F, Vielhaber S, Strigl-Pill N, Grunert B, Müller-Felber W, Glocker FX, Spranger M, Deschauer M, Mengel E, Schoser B.

J Inherit Metab Dis. 2012 Sep;35(5):837-45. Epub 2012 Jan 31.

PMID:
22290025
8.

Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study.

Kuperus E, Kruijshaar ME, Wens SCA, de Vries JM, Favejee MM, van der Meijden JC, Rizopoulos D, Brusse E, van Doorn PA, van der Ploeg AT, van der Beek NAME.

Neurology. 2017 Dec 5;89(23):2365-2373. doi: 10.1212/WNL.0000000000004711. Epub 2017 Nov 8.

PMID:
29117951
9.

Cardiopulmonary exercise test to quantify enzyme replacement response in pediatric Pompe disease.

Bar-Yoseph R, Mandel H, Mainzer G, Gur M, Tal G, Shalloufeh G, Bentur L.

Pediatr Pulmonol. 2018 Mar;53(3):366-373. doi: 10.1002/ppul.23830. Epub 2018 Jan 22.

PMID:
29356433
10.

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; POM-001/002 Investigators.

Orphanet J Rare Dis. 2017 Aug 24;12(1):144. doi: 10.1186/s13023-017-0693-2.

11.

Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease.

Rafael Bretón Martínez J, Martínez AC.

J Neurol Sci. 2015 Nov 15;358(1-2):459-60. doi: 10.1016/j.jns.2015.08.007. Epub 2015 Aug 7. No abstract available.

PMID:
26279333
12.

Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years.

Stepien KM, Hendriksz CJ, Roberts M, Sharma R.

Mol Genet Metab. 2016 Apr;117(4):413-8. doi: 10.1016/j.ymgme.2016.01.013. Epub 2016 Feb 4.

PMID:
26873529
13.

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.

Kanters TA, van der Ploeg AT, Kruijshaar ME, Rizopoulos D, Redekop WK, Rutten-van Mӧlken MPMH, Hakkaart-van Roijen L.

Orphanet J Rare Dis. 2017 Dec 13;12(1):179. doi: 10.1186/s13023-017-0731-0.

14.

24-months results in two adults with Pompe disease on enzyme replacement therapy.

Vielhaber S, Brejova A, Debska-Vielhaber G, Kaufmann J, Feistner H, Schoenfeld MA, Awiszus F.

Clin Neurol Neurosurg. 2011 Jun;113(5):350-7. doi: 10.1016/j.clineuro.2010.09.016. Epub 2011 Apr 7.

PMID:
21477922
15.

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa.

de Vries JM, van der Beek NA, Kroos MA, Ozkan L, van Doorn PA, Richards SM, Sung CC, Brugma JD, Zandbergen AA, van der Ploeg AT, Reuser AJ.

Mol Genet Metab. 2010 Dec;101(4):338-45. doi: 10.1016/j.ymgme.2010.08.009. Epub 2010 Aug 14.

PMID:
20826098
16.

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy.

Parini R, De Lorenzo P, Dardis A, Burlina A, Cassio A, Cavarzere P, Concolino D, Della Casa R, Deodato F, Donati MA, Fiumara A, Gasperini S, Menni F, Pagliardini V, Sacchini M, Spada M, Taurisano R, Valsecchi MG, Di Rocco M, Bembi B.

Orphanet J Rare Dis. 2018 Feb 8;13(1):32. doi: 10.1186/s13023-018-0771-0.

17.

An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions.

El-Gharbawy AH, Mackey J, DeArmey S, Westby G, Grinnell SG, Malovrh P, Conway R, Kishnani PS.

Mol Genet Metab. 2011 Sep-Oct;104(1-2):118-22. doi: 10.1016/j.ymgme.2011.07.004. Epub 2011 Jul 13.

18.

Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.

Schoser B, Stewart A, Kanters S, Hamed A, Jansen J, Chan K, Karamouzian M, Toscano A.

J Neurol. 2017 Apr;264(4):621-630. doi: 10.1007/s00415-016-8219-8. Epub 2016 Jul 2. Review.

PMID:
27372449
19.

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.

Masat E, Laforêt P, De Antonio M, Corre G, Perniconi B, Taouagh N, Mariampillai K, Amelin D, Mauhin W, Hogrel JY, Caillaud C, Ronzitti G, Puzzo F, Kuranda K, Colella P, Mallone R, Benveniste O, Mingozzi F; French Pompe Registry Study Group.

Sci Rep. 2016 Nov 4;6:36182. doi: 10.1038/srep36182.

20.

Early higher dosage of alglucosidase alpha in classic Pompe disease.

Spada M, Pagliardini V, Ricci F, Biamino E, Mongini T, Porta F.

J Pediatr Endocrinol Metab. 2018 Dec 19;31(12):1343-1347. doi: 10.1515/jpem-2018-0336.

PMID:
30433875

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