P. Young

发表

A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A

P. van Damme, David John Adams, J. Statland, 2021, Orphanet Journal of Rare Diseases.

An integrative correlation of myopathology, phenotype and genotype in late onset Pompe disease

K. Claeys, W. Stenzel, C. Mawrin, 2020, Neuropathology and applied neurobiology.

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascend

B. Byrne, A. Pestronk, P. van Damme, 2019, Neuromuscular Disorders.

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

A. Pestronk, C. Bjartmar, R. Carlier, 2016, Molecular genetics and metabolism.

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

M. Tarnopolsky, Y. Chien, J. Charrow, 2021, The Lancet Neurology.

Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature

M. Polkey, H. Tiddens, D. Orlikowski, 2017, Orphanet Journal of Rare Diseases.

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease

B. Byrne, P. van Damme, R. Barohn, 2022, Neurology.

The nature of respiratory muscle weakness in patients with late-onset Pompe disease

W. Randerath, D. Görlich, M. Boentert, 2019, Neuromuscular Disorders.

Pulmonary function tests (maximum inspiratory pressure, maximum expiratory pressure, vital capacity, forced vital capacity) predict ventilator use in late-onset Pompe disease

K. Berger, T. Mozaffar, A. Quartel, 2016, Neuromuscular Disorders.

Diagnose und Therapie des Late-onset-Morbus-Pompe

M. Vorgerd, B. Schoser, Z. Lukacs, 2013, Der Nervenarzt.

Use of peripheral electrical stimulation on healthy individual and patients after stroke and its effects on the somatosensory evoked potentials. A systematic review

B. Schoser, M. Mijić, P. Young, 2022, Frontiers in Neurology.

Efficacy of functional electrical stimulation in rehabilitating patients with foot drop symptoms after stroke and its correlation with somatosensory evoked potentials—a crossover randomised controlled trial

B. Schoser, P. Young, M. Mijic, 2022, Neurological Sciences.

Polyneuropathies. Etiology, Diagnosis, and Treatment Options

C. Sommer, B. Schoser, R. Forst, 2018 .

Sleep‐related symptoms and sleep‐disordered breathing in adult Pompe disease

B. Schoser, N. Karabul, S. Wenninger, 2015, European journal of neurology.

[Diagnosis and therapy of late onset Pompe disease].

M. Vorgerd, B. Schoser, Z. Lukacs, 2013, Der Nervenarzt.

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

A. Pestronk, C. Bjartmar, R. Carlier, 2016, Molecular genetics and metabolism.

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascend

B. Byrne, A. Pestronk, P. van Damme, 2019, Neuromuscular Disorders.