Mária Judit Molnár

Discovery of I-BRD9, a Selective Cell Active Chemical Probe for Bromodomain Containing Protein 9 Inhibition

Natalie H. Theodoulou, Paul Bamborough, Andrew J. Bannister et al. · Journal of Medicinal Chemistry · 2015 · 229 citations · Full text

Chemical Biology, Chromatin, Molecular Characterization +14

<i>TARDBP</i> variation associated with frontotemporal dementia, supranuclear gaze palsy, and chorea

Gábor G. Kovács, Jill R. Murrell, Sandor Horvath et al. · Movement Disorders · 2009 · 201 citations

Neuropsychology, Motor Neuron Disease, Genetics +17

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

Jordi Díaz‐Manera, Priya S. Kishnani, Hani Kushlaf et al. · The Lancet Neurology · 2021 · 169 citations · Full text

Late-onset Pompe Disease, Autoimmune Disease, Clinical Infectious Disease +3

Factors Influencing the Efficacy, Longevity, and Safety of Electroporation-Assisted Plasmid-Based Gene Transfer into Mouse Muscles

Mária Judit Molnár, Rénald Gilbert, Yifan Lu et al. · Molecular Therapy · 2004 · 141 citations · Full text

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Benedikt Schoser, Mark Roberts, Barry J. Byrne et al. · The Lancet Neurology · 2021 · 124 citations

Clinical Trials, Cipaglucosidase Alfa, Pharmacotherapy +2