Simone Weiss

Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy

Astrid Pechmann, Max Behrens, Katharina Dörnbrack et al. · Brain · 2022 · 70 citations · Full text

Medicine, Neurology, Pediatrics +15

Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial

Janbernd Kirschner, Joachim Schessl, Ulrike Schara et al. · The Lancet Neurology · 2010 · 65 citations

Medicine, Pharmacotherapy, Clinical Pharmacology +3

A genome-wide CRISPR-Cas9 knockout screen identifies novel PARP inhibitor resistance genes in prostate cancer

Malene Blond Ipsen, Ea Marie Givskov Sørensen, Emil Aagaard Thomsen et al. · Oncogene · 2022 · 32 citations

Life Sciences, Molecular Biology, Oncology +16

Biallelic loss-of-function P4HTM gene variants cause hypotonia, hypoventilation, intellectual disability, dysautonomia, epilepsy, and eye abnormalities (HIDEA syndrome)

Elisa Rahikkala, Matti Myllykoski, Reetta Hinttala et al. · Genetics in Medicine · 2019 · 26 citations · Full text

Life Sciences, Genetics, Molecular Genetics +15

Up-Regulated FGFR1 Expression as a Mediator of Intrinsic TKI Resistance in EGFR-Mutated NSCLC

Kristine Raaby Gammelgaard, Johan Vad‐Nielsen, Michelle Simone Clement et al. · Translational Oncology · 2018 · 24 citations · Full text

Medicine, Oncology, Pharmacology +17