H. Kletzl

发表

Functional improvement in mouse models of familial amyotrophic lateral sclerosis by PEGylated insulin-like growth factor I treatment depends on disease severity

F. Metzger, A. Hoeflich, W. Spooren, 2012, Amyotrophic lateral sclerosis : official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases.

706 POSTER A phase I dose escalation pharmacokinetic (PK) and pharmacodynamic (PD) study of weekly and twice weekly erlotinib in advanced stage solid malignancies

K. Gelmon, S. Chia, C. Kollmannsberger, 2007 .

Bioavailability and pharmacokinetic profile of balovaptan, a selective, brain-penetrant vasopressin 1a receptor antagonist, in healthy volunteers

L. Squassante, A. Guenther, Michael G. M. Derks, 2021, Expert opinion on investigational drugs.

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

T. Bergauer, O. Khwaja, K. Heinig, 2018, British journal of clinical pharmacology.

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

G. Comi, C. Palermo, M. Pane, 2022, The Lancet Neurology.

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.

P. Fontoura, L. Servais, C. Bruno, 2021, The New England journal of medicine.

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

G. Comi, S. Corti, E. Mazzone, 2022, The Lancet Neurology.

First-in-man study with a novel PEGylated recombinant human insulin-like growth factor-I.

F. Metzger, J. Frystyk, A. Guenther, 2017, Growth hormone & IGF research : official journal of the Growth Hormone Research Society and the International IGF Research Society.

P.375 Clinical studies of RG7916 in patients with spinal muscular atrophy: SUNFISH part 1 study update

E. Mercuri, L. Servais, N. Goemans, 2017, Neuromuscular Disorders.

The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy

E. Mercuri, Hanns Lochmüller, T. Bugawan, 2019, Neuromuscular Disorders.

Risdiplam in Type 1 Spinal Muscular Atrophy.

E. Mercuri, P. Fontoura, L. Servais, 2021, The New England journal of medicine.

Ocrelizumab in relapsing and primary progressive multiple sclerosis: Pharmacokinetic and pharmacodynamic analyses of OPERA I, OPERA II and ORATORIO

L. Kappos, S. Hauser, A. Günther, 2020, British journal of clinical pharmacology.

Randomized phase II trial of erlotinib versus temozolomide or carmustine in recurrent glioblastoma: EORTC brain tumor group study 26034.

M. J. van den Bent, M. Taphoorn, A. Brandes, 2009, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

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

Michael Reutlinger, Andreas Zeller, Stefan Kustermann, 2018, Journal of medicinal chemistry.

Effect of gastric pH on erlotinib pharmacokinetics in healthy individuals: omeprazole and ranitidine

B. Lum, M. Abt, M. Giraudon, 2015, Anti-cancer drugs.

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

E. Mazzone, P. Fontoura, L. Servais, 2023, Journal of Neurology.

Effect of mild or moderate hepatic impairment on the pharmacokinetics of risdiplam

T. Marbury, K. Heinig, A. Günther, 2022, British journal of clinical pharmacology.

Addressing Today’s Absorption, Distribution, Metabolism, and Excretion (ADME) Challenges in the Translation of In Vitro ADME Characteristics to Humans: A Case Study of the SMN2 mRNA Splicing Modifier Risdiplam

Cordula Stillhart, A. Guenther, K. Heinig, 2021, Drug Metabolism and Disposition.

Early pediatric formulation development with new chemical entities: Opportunities of e-tongue besides human taste assessment.

A. Günther, S. Sturm, H. Kletzl, 2017, International journal of pharmaceutics.

Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo‐controlled, dose‐finding trial followed by 24 months of treatment

E. Mazzone, L. Servais, O. Boespflug-Tanguy, 2022, European journal of neurology.

Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies

F. Metzger, O. Khwaja, C. Czech, 2017 .

Erlotinib in combination with pemetrexed for patients with advanced non-small-cell lung cancer (NSCLC): a phase I dose-finding study.

M. Ranson, I. Melezínek, G. Klingelschmitt, 2010, Annals of oncology : official journal of the European Society for Medical Oncology.

Model‐Based Drug–Drug Interaction Extrapolation Strategy From Adults to Children: Risdiplam in Pediatric Patients With Spinal Muscular Atrophy

L. Aarons, A. Galetin, K. Ogungbenro, 2021, Clinical pharmacology and therapeutics.

Association of Higher Ocrelizumab Exposure With Reduced Disability Progression in Multiple Sclerosis

L. Kappos, A. Bar-Or, S. Hauser, 2023, Neurology: Neuroimmunology & Neuroinflammation.

Pharmacokinetics, Pharmacodynamics and Exposure-Response Analyses of Ocrelizumab in Patients With Multiple Sclerosis (N4.001)

L. Kappos, S. Hauser, A. Guenther, 2019 .

A Single Dose, Randomized, Controlled Proof-Of-Mechanism Study of a Novel Vasopressin 1a Receptor Antagonist (RG7713) in High-Functioning Adults with Autism Spectrum Disorder

E. Hollander, F. Shic, J. McCracken, 2017, Neuropsychopharmacology.

P.353FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with type 1 spinal muscular atrophy

E. Mercuri, L. Servais, G. Baranello, 2019, Neuromuscular Disorders.

A Single Dose, Randomized, Controlled Proof-Of-Mechanism Study of a Novel Vasopressin 1a Receptor Antagonist (RG7713) in High-Functioning Adults with Autism Spectrum Disorder

E. Hollander, F. Shic, J. McCracken, 2016, Neuropsychopharmacology.

A UGT2B10 Splicing Polymorphism Common in African Populations May Greatly Increase Drug Exposure

M. Finel, O. Spleiss, M. Hoener, 2015, The Journal of Pharmacology and Experimental Therapeutics.

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

R. Finkel, M. Pane, A. Mayhew, 2023, Neurology and Therapy.

FP.24 RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)

E. Bertini, E. Zanoteli, M. Farrar, 2022, Neuromuscular Disorders.

Addressing Today ’ s Absorption, Distribution, Metabolism, and Excretion (ADME) Challenges in the Translation of In Vitro ADME Characteristics to Humans: A Case Study of the SMN2 mRNA Splicing Modifier Risdiplam S

Cordula Stillhart, D. Tuerck, A. Poirier, 2021 .

Supplementary Materials for: Model-based drug-drug interaction extrapolation strategy from adults to children –risdiplam in pediatric patients with spinal muscular atrophy

L. Aarons, A. Galetin, K. Ogungbenro, 2021 .

P.064 FIREFISH Part 1: 1-year results on motor function in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916)

E. Mercuri, L. Servais, O. Khwaja, 2019, Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques.