Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency
Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Pajno, Roberta, Barzaghi, Federica, Giannelli, Stefania, Dionisio, Francesca, Brigida, Immacolata, Bonopane, Marco, Casiraghi, Miriam, Tabucchi, Antonella, Carlucci, Filippo, Grunebaum, Eyal, Adeli, Mehdi, Bredius, Robbert G., Puck, Jennifer M., Stepensky, Polina, Tezcan, Ilhan, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Ciceri, Fabio, Roncarolo, Maria Grazia, Aiuti, Alessandro
Published in Blood (07.07.2016)
Published in Blood (07.07.2016)
Get full text
Journal Article
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
Voit, Thomas, Prof, Topaloglu, Haluk, Prof, Straub, Volker, Prof, Muntoni, Francesco, Prof, Deconinck, Nicolas, MD, Campion, Giles, MD, De Kimpe, Sjef J, PhD, Eagle, Michelle, PhD, Guglieri, Michela, MD, Hood, Steve, PhD, Liefaard, Lia, PhD, Lourbakos, Afrodite, PhD, Morgan, Allison, BSc, Nakielny, Joanna, MB, Quarcoo, Naashika, PhD, Ricotti, Valeria, MB, Rolfe, Katie, MSc, Servais, Laurent, MD, Wardell, Claire, DPhil, Wilson, Rosamund, PhD, Wright, Padraig, MD, Kraus, John E, MD
Published in Lancet neurology (01.10.2014)
Published in Lancet neurology (01.10.2014)
Get full text
Journal Article
Optimal balance of benefit versus risk for tafenoquine in the treatment of Plasmodium vivax malaria
Sharma, Raman, Sharma, Hema, Jones, Siôn, Borghini-Fuhrer, Isabelle, Domingo, Gonzalo J, Gibson, Rachel A, Rolfe, Katie, Tan, Lionel, Fiţa, Ioana Gabriela, Chen, Chao, Bird, Panayota, Pingle, Anup, Duparc, Stephan
Published in Malaria journal (13.05.2024)
Published in Malaria journal (13.05.2024)
Get full text
Journal Article
Comment on 'The clinical pharmacology of tafenoquine in the radical cure of Plasmodium vivax malaria: An individual patient data meta-analysis'
Sharma, Raman, Chen, Chao, Tan, Lionel, Rolfe, Katie, Fiţa, Ioana-Gabriela, Jones, Siôn, Pingle, Anup, Gibson, Rachel A, Goyal, Navin, Sharma, Hema, Bird, Panayota
Published in eLife (07.02.2024)
Published in eLife (07.02.2024)
Get full text
Journal Article
A Retrospective, Pooled Suicidality Evaluation of Ropinirole Immediate Release and Controlled Release for the Treatment of Restless Legs Syndrome
Makumi, Clare W., PharmD, MBA, Paska, Walter, PhD, Rolfe, Katie, MSc, Shulman, Kenneth J., DO
Published in Clinical therapeutics (01.05.2015)
Published in Clinical therapeutics (01.05.2015)
Get full text
Journal Article
Sleep Duration and "on" Time during Different Periods of the Day and Night in Patients with Advanced Parkinson's Disease Receiving Adjunctive Ropinirole Prolonged Release
Reichmann, Heinz, Cooper, James, Rolfe, Katie, Martinez-Martin, Pablo
Published in Parkinson's disease (01.01.2011)
Published in Parkinson's disease (01.01.2011)
Get full text
Journal Article
Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety
Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Roncarolo, Maria Grazia, Aiuti, Alessandro
Published in Molecular therapy (07.03.2018)
Published in Molecular therapy (07.03.2018)
Get full text
Journal Article
A pharmacometrics approach to assess the feasibility of capillary microsampling to replace venous sampling in clinical studies: Tafenoquine case study
Bachhav, Sagar S., Taylor, Maxine, Martin, Ana, Green, Justin A., Duparc, Stephan, Rolfe, Katie, Sharma, Hema, Tan, Lionel K., Goyal, Navin
Published in British journal of clinical pharmacology (01.03.2023)
Published in British journal of clinical pharmacology (01.03.2023)
Get full text
Journal Article
Tafenoquine co-administered with dihydroartemisinin–piperaquine for the radical cure of Plasmodium vivax malaria (INSPECTOR): a randomised, placebo-controlled, efficacy and safety study
Sutanto, Inge, Soebandrio, Amin, Ekawati, Lenny L, Chand, Krisin, Noviyanti, Rintis, Satyagraha, Ari Winasti, Subekti, Decy, Santy, Yulia Widya, Crenna-Darusallam, Chelzie, Instiaty, Instiaty, Budiman, Waras, Prasetya, Catur Bidik, Lardo, Soroy, Elyazar, Iqbal, Duparc, Stephan, Cedar, Eve, Rolfe, Katie, Fernando, Disala, Berni, Alessandro, Jones, Siôn, Kleim, Jörg-Peter, Fletcher, Kim, Sharma, Hema, Martin, Ana, Taylor, Maxine, Goyal, Navin, Green, Justin A, Tan, Lionel K, Baird, J Kevin
Published in The Lancet infectious diseases (01.10.2023)
Published in The Lancet infectious diseases (01.10.2023)
Get full text
Journal Article
Tafenoquine for the radical cure versus prophylaxis of Plasmodium vivax malaria: the importance of using the appropriate data set
Sharma, Raman, Chen, Chao, Fiţa, Ioana-Gabriela, Gibson, Rachel, Jones, Siôn, Pingle, Anup, Rolfe, Katie, Sharma, Hema, Bird, Panayota
Published in Indian journal of medical research (New Delhi, India : 1994) (01.08.2023)
Published in Indian journal of medical research (New Delhi, India : 1994) (01.08.2023)
Get full text
Journal Article
A human microdose study of the antimalarial drug GSK3191607 in healthy volunteers
Okour, Malek, Derimanov, Geo, Barnett, Rodger, Fernandez, Esther, Ferrer, Santiago, Gresham, Stephanie, Hossain, Mohammad, Gamo, Francisco‐Javier, Koh, Gavin, Pereira, Adrian, Rolfe, Katie, Wong, Deborah, Young, Graeme, Rami, Harshad, Haselden, John
Published in British journal of clinical pharmacology (01.03.2018)
Published in British journal of clinical pharmacology (01.03.2018)
Get full text
Journal Article
Application of the Stable Isotope Label Approach in Clinical Development—Supporting Dissolution Specifications for a Commercial Tablet Product with Tafenoquine, a Long Half-life Compound
Goyal, Navin, Mohamed, Khadeeja, Rolfe, Katie, Sahota, Satty, Ernest, Terry, Duparc, Stephan, Taylor, Maxine, Casillas, Linda, Koh, Gavin C. K. W.
Published in The AAPS journal (01.07.2018)
Published in The AAPS journal (01.07.2018)
Get full text
Journal Article
Tafenoquine exposure assessment, safety, and relapse prevention efficacy in children with Plasmodium vivax malaria: open-label, single-arm, non-comparative, multicentre, pharmacokinetic bridging, phase 2 trial
Vélez, Iván D, Hien, Tran T, Green, Justin A, Martin, Ana, Sharma, Hema, Rousell, Victoria M, Breton, John J, Ernest, Terry B, Rolfe, Katie, Taylor, Maxine, Mohamed, Khadeeja, Jones, Siôn W, Chau, Nguyen Hoang, Hoa, Nhu Thi, Duparc, Stephan, Tan, Lionel K, Goyal, Navin
Published in The lancet child & adolescent health (01.02.2022)
Published in The lancet child & adolescent health (01.02.2022)
Get full text
Journal Article
A Flexible Multi-Metric Bayesian Framework for Decision-Making in Phase II Multi-Arm Multi-Stage Studies
Dufault, Suzanne M, Crook, Angela M, Rolfe, Katie, Phillips, Patrick P J
Published in arXiv.org (23.10.2023)
Published in arXiv.org (23.10.2023)
Get full text
Paper
Journal Article
Sleep Duration and “on” Time during Different Periods of the Day and Night in Patients with Advanced Parkinson's Disease Receiving Adjunctive Ropinirole Prolonged Release
Reichmann, Heinz, Cooper, James, Rolfe, Katie, Martinez-Martin, Pablo
Published in Pathology research international (2011)
Get full text
Published in Pathology research international (2011)
Journal Article