Heidemarie Kletzl

Randomized Phase II Trial of Erlotinib Versus Temozolomide or Carmustine in Recurrent Glioblastoma: EORTC Brain Tumor Group Study 26034

Martin J. van den Bent, Alba A. Brandes, R. Rampling et al. · Journal of Clinical Oncology · 2009 · 561 citations · Full text

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( <i>SMN2</i> ) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Hasane Ratni, Martin Ebeling, John D. Baird et al. · Journal of Medicinal Chemistry · 2018 · 492 citations · Full text

A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a <i>SMN2</i> splicing modifier

Stefan Sturm, Andreas Guenther, Birgit Jaber et al. · British Journal of Clinical Pharmacology · 2018 · 98 citations · Full text

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose et al. · The Lancet Neurology · 2022 · 96 citations · Full text

Type 1, Firefish Part 2, Rehabilitation +6

Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)

Maryam Oskoui, John Day, Nicolas Deconinck et al. · Journal of Neurology · 2023 · 85 citations · Full text