NEO1 and NEO-EXT studies: Long-term safety of repeat avalglucosidase alfa dosing for 4.5 years in late-onset Pompe disease patients

Pena, Loren; Barohn, Richard J; Dimachkie, Mazen M; Kishnani, Priya S; Ladha, Shafeeq; Mengel, Karl-Eugen; Sacconi, Sabrina; Van Damme, Philip; Vissing, John; Haack, Kristina An; Hug, Christopher; Johnson, Judith; Sensinger, Charlotte; Schoser, Benedikt

doi:10.1016/j.ymgme.2018.12.294

Molecular Genetics and Metabolism

NEO1 and NEO-EXT studies: Long-term safety of repeat avalglucosidase alfa dosing for 4.5 years in late-onset Pompe disease patients

Author:

Pena, Loren

Barohn, Richard J ; Dimachkie, Mazen M ; Kishnani, Priya S ; Ladha, Shafeeq ; Mengel, Karl-Eugen ; Sacconi, Sabrina ; Van Damme, Philip ; Vissing, John ; Haack, Kristina An ; Hug, Christopher ; Johnson, Judith ; Sensinger, Charlotte ; Schoser, Benedikt

Keywords:

Science & Technology, Life Sciences & Biomedicine, Endocrinology & Metabolism, Genetics & Heredity, Medicine, Research & Experimental, Research & Experimental Medicine, 1103 Clinical Sciences, 3105 Genetics, 3202 Clinical sciences

Molecular Genetics and Metabolism NEO1 and NEO-EXT studies: Long-term safety of repeat avalglucosidase alfa dosing for 4.5 years in late-onset Pompe disease patients

Author:

Keywords:

Molecular Genetics and Metabolism

NEO1 and NEO-EXT studies: Long-term safety of repeat avalglucosidase alfa dosing for 4.5 years in late-onset Pompe disease patients