Phenotypic continuum in neuronopathic gaucher disease: an intermediate phenotype between type 2 and type 3
Goker-Alpan, Ozlem, Schiffmann, Raphael, Park, Joseph K, Stubblefield, Barbara K, Tayebi, Nahid, Sidransky, Ellen
Published in The Journal of pediatrics (01.08.2003)
Published in The Journal of pediatrics (01.08.2003)
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T.P.24
Thurberg, B.L, van der Ploeg, A, Kissel, J.T, Schoser, B, Pestronk, A, Barohn, R.J, Goker-Alpan, O, Mozaffar, T, Pena, L.D.M, Simmons, Z, Straub, V, Young, P, Bjartmar, C
Published in Neuromuscular disorders : NMD (01.10.2014)
Published in Neuromuscular disorders : NMD (01.10.2014)
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T.P.46 A novel phase 2a study design to investigate drug-drug interactions between escalating doses of AT2220 (duvoglustat hydrochloride) and acid alpha-glucosidase in subjects with Pompe disease
Kishnani, P, Tarnopolsky, M, Sivakumar, K, Byrne, B, Goker-Alpan, O, Guter, K, Pervaiz, M, Dasouki, M, Levine, T, Roberts, M, Johnson, F.K, Valenzano, K.J, Lockhart, D.J, Boudes, P
Published in Neuromuscular disorders : NMD (01.10.2012)
Published in Neuromuscular disorders : NMD (01.10.2012)
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T.P.45 An ongoing phase 2a study to investigate drug–drug interactions between escalating doses of AT2220 (duvoglustat hydrochloride) and acid alpha glucosidase in subjects with Pompe disease – Preliminary results
Kishnani, P, Tarnopolsky, M, Sivakumar, K, Byrne, B, Goker-Alpan, O, Guter, K, Pervaiz, M, Dasouki, M, Levine, T, Roberts, M, Johnson, F.K, Sitaraman, S, Khanna, R, Flanagan, J, Sjoberg, E.R, Valenzano, K.J, Lockhart, D.J, Boudes, P
Published in Neuromuscular disorders : NMD (01.10.2012)
Published in Neuromuscular disorders : NMD (01.10.2012)
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Clinical Outcomes With Recombinant Human C1 Inhibitor In The Repeat Treatment Of Acute Attacks Of Hereditary Angioedema In North-american Patients
Zuraw, B, Craig, T, Lockey, R.F, Park, M, Grant, J, Suez, D, Schwartz, L.B, Riedl, M, Levy, R.J, Li, H.H, Edara, L, Bernstein, J.A, Moy, J, Offenberger, J, Szema, A, Engler, A, Rehman, S.M, Marbury, T.C, Alpan, O, Bahna, S.L, Davis, A, Sussman, G, Yang, W.H, Ritchie, B, Relan, A
Published in Journal of allergy and clinical immunology (01.02.2011)
Published in Journal of allergy and clinical immunology (01.02.2011)
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P372 Safety of home administration of cipaglucosidase alfa + miglustat in late-onset Pompe disease: results from multiple clinical trials
Byrne, B., Diaz-Manera, J., Goker-Alpan, O., Mozaffar, T., Wasfi, Y., Das, S Sitaraman, Fox, B., Holdbrook, F., Jain, V.
Published in Neuromuscular disorders : NMD (01.10.2023)
Published in Neuromuscular disorders : NMD (01.10.2023)
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FP.17 Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)
Schoser, B., Kishnani, P., Bratkovic, D., Clemens, P., Goker-Alpan, O., Ming, X., Roberts, M., Vorgerd, M., Sivakumar, K., van der Ploeg, A., Goldman, M., Wright, J., Holdbrook, F., Jain, V., Sitaraman, S., Wasfi, Y., Mozaffar, T., Byrne, B.
Published in Neuromuscular disorders : NMD (01.10.2022)
Published in Neuromuscular disorders : NMD (01.10.2022)
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Glucocerebrosidase mutations in subjects with parkinsonism
Lwin, Alicia, Orvisky, Eduard, Goker-Alpan, Ozlem, LaMarca, Mary E, Sidransky, Ellen
Published in Molecular genetics and metabolism (2004)
Published in Molecular genetics and metabolism (2004)
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PRECLINICAL APPROACHES AND EARLY CLINICAL RESULTS: O.22First-in-human study of ATB200/AT2221 in patients with Pompe disease: 24-month functional assessment results from the ATB200-02 trial
Schoser, B., Kishnani, P., Bratkovic, D., Byrne, B., Clemens, P., Goker-Alpan, O., Ming, X., Roberts, M., Schwenkreis, P., Sivakumar, K., van der Ploeg, A., Sitaraman, S., Barth, J., Lagast, H., Mozaffar, T.
Published in Neuromuscular disorders : NMD (01.10.2019)
Published in Neuromuscular disorders : NMD (01.10.2019)
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PRECLINICAL APPROACHES AND EARLY CLINICAL RESULTS
Schoser, B., Kishnani, P., Bratkovic, D., Byrne, B., Clemens, P., Goker-Alpan, O., Ming, X., Roberts, M., Schwenkreis, P., Sivakumar, K., van der Ploeg, A., Sitaraman, S., Barth, J., Lagast, H., Mozaffar, T.
Published in Neuromuscular disorders : NMD (01.10.2019)
Published in Neuromuscular disorders : NMD (01.10.2019)
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AUTOPHAGIC MYOPATHIES / MYOFIBRILLAR MYOPATHIES / DISTAL MYOPATHIES / POMPE DISEASE
Schoser, B., Barohn, R., Byrne, B., Goker-Alpan, O., Kishnani, P., Ladha, S., Laforet, P., Mengel, E., Pena, L., Sacconi, S., Straub, V., Trivedi, J., Van Damme, P., van der Ploeg, A., Vissing, J., Young, P., Haack, K., Ivanina, I., Wang, Y., Dimachkie, M.
Published in Neuromuscular disorders : NMD (01.10.2020)
Published in Neuromuscular disorders : NMD (01.10.2020)
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AUTOPHAGIC MYOPATHIES / MYOFIBRILLAR MYOPATHIES / DISTAL MYOPATHIES / POMPE DISEASE: P.03 NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in late-onset Pompe disease (LOPD)
Schoser, B., Barohn, R., Byrne, B., Goker-Alpan, O., Kishnani, P., Ladha, S., Laforet, P., Mengel, E., Pena, L., Sacconi, S., Straub, V., Trivedi, J., Van Damme, P., van der Ploeg, A., Vissing, J., Young, P., Haack, K., Ivanina, I., Wang, Y., Dimachkie, M.
Published in Neuromuscular disorders : NMD (01.10.2020)
Published in Neuromuscular disorders : NMD (01.10.2020)
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NEW THERAPEUTIC APPROACHES AND THEIR READOUT
Schoser, B., Bratkovic, D., Byrne, B., Clemens, P., Geberhiwot, T., Goker-Alpan, O., Kishnani, P., Ming, X., Mozaffar, T., Schwenkreis, P., Sivakumar, K., van der Ploeg, A., Wright, J., Johnson, F., Sitaraman, S., Barth, J., Sathe, S., Roberts, M.
Published in Neuromuscular disorders : NMD (01.10.2018)
Published in Neuromuscular disorders : NMD (01.10.2018)
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NEW THERAPEUTIC APPROACHES AND THEIR READOUT: O.20Results from ATB200-02: first-in-human, open-label, phase 1/2 study of ATB200 co-administered with AT2221 for Pompe disease
Schoser, B., Bratkovic, D., Byrne, B., Clemens, P., Geberhiwot, T., Goker-Alpan, O., Kishnani, P., Ming, X., Mozaffar, T., Schwenkreis, P., Sivakumar, K., van der Ploeg, A., Wright, J., Johnson, F., Sitaraman, S., Barth, J., Sathe, S., Roberts, M.
Published in Neuromuscular disorders : NMD (01.10.2018)
Published in Neuromuscular disorders : NMD (01.10.2018)
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P.69NEO1 and NEO-EXT studies: exploratory efficacy of repeat avalglucosidase alfa dosing for up to 5 years in participants with late-onset Pompe disease (LOPD)
Schoser, B., Barohn, R., Byrne, B., Goker-Alpan, O., Kishnani, P., Ladha, S., Laforêt, P., Mengel, K., Pena, L., Sacconi, S., Straub, V., Trivedi, J., Van Damme, P., van der Ploeg, A., Vissing, J., Young, P., An Haack, K., Liu, K., Fraser, P., Dimachkie, M.
Published in Neuromuscular disorders : NMD (01.10.2019)
Published in Neuromuscular disorders : NMD (01.10.2019)
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T.P.24: Alglucosidase alfa reduces lysosomal glycogen in skeletal muscle biopsies of patients with late-onset Pompe disease (LOPD)
Thurberg, B.L., van der Ploeg, A., Kissel, J.T., Schoser, B., Pestronk, A., Barohn, R.J., Goker-Alpan, O., Mozaffar, T., Pena, L.D.M., Simmons, Z., Straub, V., Young, P., Bjartmar, C.
Published in Neuromuscular disorders : NMD (01.10.2014)
Published in Neuromuscular disorders : NMD (01.10.2014)
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