Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials

Authors: Díaz-Manera, Jordi ^{a, ⁎}, Hughes, Derralynn ^b, Erdem-Özdamar, Sevim ^c, Tard, Céline ^d, Béhin, Anthony ^e, Bouhour, Françoise ^f, Davison, James ^{g, m}, Hahn, Si Houn ^h, Haack, Kristina An ⁱ, Huynh-Ba, Olivier ⁱ, Periquet, Magali ^{j, 1}, Tammireddy, Swathi ^k, Thibault, Nathan ^k, Zhou, Tianyue ^k, van der Ploeg, Ans T. ^l

Source: In Molecular Genetics and Metabolism December 2024 143(4)

Efficacy and safety of avalglucosidase alfa in participants with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

Authors: Kishnani, Priya S., Díaz-Manera, Jordi, Kushlaf, Hani, Ladha, Shafeeq, Mozaffar, Tahseen, Straub, Volker, Toscano, Antonio, van der Ploeg, Ans T., Clemens, Paula R., Day, John W., Illarioshkin, Sergey, Roberts, Mark E., Attarian, Shahram, Carvalho, Gerson, Erdem-Özdamar, Sevim, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, Haack, Kristina An, Huynh-Ba, Olivier, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M., Schoser, Benedikt

Source: Molecular genetics and metabolism. 138(2)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100180903087.0x000001&indx=1&recIds=ETOCvdc_100180903087.0x000001

Mini-COMET study: Effects of 97 weeks of avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease who were previously treated with alglucosidase alfa

Authors: Kishnani, Priya S., Weinert, Marguerite C., Davison, James, Broomfield, Alexander, Chien, Yin-Hsiu, Hahn, Sihoun, Kronn, David, Kumada, Satoko, Ohki, Hirotaka, Pichard, Samia, Haack, Kristina An, Tammireddy, Swathi, Zhou, Tianyue, Prakalapakorn, Sasapin G.

Source: Molecular genetics and metabolism. 138(2)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100180903072.0x000001&indx=1&recIds=ETOCvdc_100180903072.0x000001

Corrigendum to “Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials” [Molecular Genetics and Metabolism, 143 (2024) 108608]

Authors: Díaz-Manera, Jordi ^{a, ⁎}, Hughes, Derralynn ^b, Erdem-Özdamar, Sevim ^c, Tard, Céline ^d, Béhin, Anthony ^e, Bouhour, Françoise ^f, Davison, James ^{g, m}, Hahn, Si Houn ^h, Haack, Kristina An ⁱ, Huynh-Ba, Olivier ⁱ, Periquet, Magali ^{j, 1}, Tammireddy, Swathi ^k, Thibault, Nathan ^k, Zhou, Tianyue ^k, van der Ploeg, Ans T. ^l

Source: In Molecular Genetics and Metabolism March 2025 144(3)

Mini-COMET study: Safety and efficacy data after avalglucosidase alfa dosing for ≥ 145 weeks in participants with infantile-onset Pompe disease (IOPD) who had demonstrated clinical decline or sub-optimal response while receiving alglucosidase alfa

Authors: Kronn, David, Kishnani, Priya, Davison, James, Brassier, Anais, Broomfield, Alexander, Hahn, Sihoun, Kumada, Satoko, Labarthe, François, Ohki, Hirotaka, Haack, Kristina An, Sparks, Susan, Tammireddy, Swathi, Zaher, Atef, Zhou, Tianyue, Chien, Yin-Hsiu

Source: In Molecular Genetics and Metabolism February 2024 141(2)

Using a novel measuring device to objectively evaluate the clinical benefit on ptosis in infantile-onset Pompe disease (IOPD): Results from 145 weeks' avalglucosidase alfa dosing in Mini-COMET participants previously treated with alglucosidase alfa

Authors: Kishnani, Priya S., Weinert, Marguerite C., Davison, James, Brassier, Anais, Broomfield, Alexander, Chien, Yin-Hsiu, Hahn, Sihoun, Kronn, David, Kumada, Satoko, Labarthe, François, Ohki, Hirotaka, Haack, Kristina An, Huynh-Ba, Olivier, Sparks, Susan, Tammireddy, Swathi, Zhou, Tianyue, Prakalapakorn, S. Grace

Source: In Molecular Genetics and Metabolism February 2024 141(2)

Baby-COMET: Safety of avalglucosidase alfa after repeat dosing in treatment-naïve participants with infantile-onset Pompe disease (IOPD)

Authors: Hahn, Andreas, Fu, Lijun, Gasperini, Serena, Witters, Peter, Accomando, Beverly, Haack, Kristina An, Huynh-Ba, Olivier, Sparks, Susan, Tammireddy, Swathi, Chien, Yin-Hsiu

Source: In Molecular Genetics and Metabolism February 2024 141(2)

Avalglucosidase alfa immunogenicity in alglucosidase alfa-experienced participants with Pompe disease: Pooled analysis of clinical trial data

Authors: Kishnani, Priya S., Richards, Susan, Al-Hassnan, Zuhair Nassair, Filosto, Massimiliano, Hahn, Andreas, Needham, Merrilee, Sacconi, Sabrina, van den Hout, Hannerieke, Haack, Kristina An, Miossec, Patrick, Sparks, Susan, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Broomfield, Alexander

Source: Molecular genetics and metabolism. 135(2):S67-S68

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100154953017.0x000001&indx=1&recIds=ETOCvdc_100154953017.0x000001

FGFR1 Amplification in Squamous Cell Carcinoma of The Lung

Authors: Heist, Rebecca S., Mino-Kenudson, Mari, Sequist, Lecia V., Tammireddy, Swathi, Morrissey, Laura, Christiani, David C., Engelman, Jeffrey A., Iafrate, A. John

Source: In Journal of Thoracic Oncology December 2012 7(12):1775-1780

Baby-COMET methodology: A clinical study of the efficacy, safety, pharmacokinetics, and pharmacodynamics of avalglucosidase alfa in treatment-naïve participants with IOPD

Authors: Broomfield, Alexander, Davison, James, Donati, Maria Alice, Haack, Kristina An, Tammireddy, Swathi, Thibault, Nathan, Woloschak, Michael, Zaher, Atef, Zhou, Tianyue, Hahn, Andreas

Source: Molecular genetics and metabolism. 135(2):S26-S26

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100154954166.0x000001&indx=1&recIds=ETOCvdc_100154954166.0x000001

Home-infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials (COMET, NEO-EXT, and Mini-COMET)

Authors: Díaz-Manera, Jordi, Hughes, Derralynn, Béhin, Anthony, Bouhour, Françoise, Davison, James, Erdem-Özdamar, Sevim, Hahn, Si Houn, Haack, Kristina An, Huynh-Ba, Olivier, Periquet, Magali, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Tard, Céline, van der Ploeg, Ans T.

Source: Molecular genetics and metabolism. 135(2):S38-S38

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100154953819.0x000001&indx=1&recIds=ETOCvdc_100154953819.0x000001

Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline

Authors: Kronn, David, Davison, James, Brassier, Anaïs, Broomfield, Alexander, Hahn, Si Houn, Kumada, Satoko, Labarthe, François, Ohki, Hirotaka, Prakalapakorn, S. Grace, Haack, Kristina An, Meng, Xianzhang, Sparks, Susan, Tammireddy, Swathi, Wilson, Catherine, Zaher, Atef, Zhou, Tianyue, Chien, Yin-Hsiu, Kishnani, Priya S.

Source: Molecular genetics and metabolism. 135(2):S68-S68

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100154952993.0x000001&indx=1&recIds=ETOCvdc_100154952993.0x000001

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

Authors: Kishnani, Priya S., Diaz-Manera, Jordi, Toscano, Antonio, Clemens, Paula R., Ladha, Shafeeq, Berger, Kenneth I., Kushlaf, Hani, Straub, Volker, Carvalho, Gerson, Mozaffar, Tahseen, Roberts, Mark, Attarian, Shahram, Chien, Yin-Hsiu, Choi, Young-Chul, Day, John W., Erdem-Ozdamar, Sevim, Illarioshkin, Sergey, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, van der Ploeg, Ans T., An Haack, Kristina, Huynh-Ba, Olivier, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M., Schoser, Benedikt

Source: JAMA Neurology; June 2023, Vol. 80 Issue: 6 p558-567, 10p

OP016: Mini-COMET: Safety and efficacy of ≥97 weeks’ avalglucosidase alfa in infantile-onset Pompe disease participants previously treated with alglucosidase alfa

Authors: Kronn, David, Davison, James, Brassier, Anaïs, Broomfield, Alexander, Hahn, Si Houn, Kumada, Satoko, Labarthe, François, Ohki, Hirotaka, Prakalapakorn, S. Grace, Haack, Kristina An, Meng, Xianzhang, Sparks, Susan, Tammireddy, Swathi, Wilson, Catherine, Zaher, Atef, Zhou, Tianyue, Chien, Yin-Hsiu, Kishnani, Priya, On behalf of the Mini-COMET investigators

Source: In Genetics in Medicine March 2022 24(3) Supplement:S348-S349

This result is not displayed to guests.
Login for full access.

This result is not displayed to guests.
Login for full access.