The Global FKRP Registry is operated by the John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom. Data is stored in the United Kingdom under the responsibility of Prof. Volker Straub. The German-speaking branch of the FKRP Registry is operated by the Friedrich-Baur-Institut, Klinikum der Universität München (University of Munich Hospital) and its data is stored at the Klinikum der Universität München under the responsibility of Prof. Maggie Walter. Data access is through defined personnel at both Klinikum der Universität München and Newcastle University.
Head of the Global FKRP Registry (international)
Volker Straub is the Deputy Dean, Harold Macmillan Professor of Medicine and Professor of Neuromuscular Genetics at the Institute of Translational and Clinical Research at Newcastle University, United Kingdom. He is the Director of the university’s John Walton Muscular Dystrophy Research Centre and holds honorary clinical appointments with the Newcastle upon Tyne Hospitals NHS Foundation Trust and the North Tees and Hartlepool NHS Foundation Trust.
Volker was trained as a paediatric neurologist at the University of Düsseldorf and the University of Essen in Germany. He wrote his PhD thesis on Duchenne muscular dystrophy (DMD) and worked as a postdoctoral research fellow in Dr Kevin Campbell’s laboratory at the Howard Hughes Medical Institute at the University of Iowa in Iowa City, Iowa, USA, on limb girdle muscular dystrophies (LGMD).
Volker has a long-standing interest in the pathogenesis of genetic muscle diseases, with research using zebrafish and mouse models. His current research also involves the application of magnetic resonance imaging, next generation sequencing and other –omics technologies for the characterization of primary neuromuscular disorders.
One of Volker’s main interests in muscle diseases is around translational research. He was the co-founder of the EU FP6 funded network of excellence for genetic neuromuscular diseases, TREAT-NMD (www.treat-nmd.eu), which he coordinated together with Kate Bushby. Volker is a long standing core member of the TREAT-NMD TACT committee (http://www.treat-nmd.eu/resources/tact/introduction/), which offers guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases.
Volker is the CI/ PI for a number of natural history and interventional trials in DMD, LGMD, Pompe disease, spinal muscular atrophy and other NMDs. Volker also partners with the Broad Institute and MIT to lead the MYO-SEQ study, a pan European next generation sequencing programme targeted at patients with limb girdle weakness of unknown origin (https://myo-seq.org). Volker recently chaired the European MYO-MRI COST Action (BM1304) to develop applications of MR imaging and spectroscopy techniques in neuromuscular disease. He is part of the steering committee of the MRC Centre for neuromuscular diseases and is now President of the World Muscle Society. He is an author on >300 peer-reviewed publications.
Head of the Global FKRP Registry (Germany)
If you would like to speak with someone about country specific issues relating to your health or the registry then please get in touch with your national contact.
Lindsay Murphy (Global)
In 2018, Lindsay Murphy joined the Patient Registries Team at the John Walton Muscular Dystrophy Research Centre, Newcastle University, UK. She coordinates both the Global FKRP Registry and the UK SMA Patient Registry. She is the first point of contact for the Global FKRP Registry and welcomes questions and comments from patients and their families and encourages applications for use of the registry by patient, researcher, academic or industry stakeholders working to improve the knowledge, understanding, care and treatment of FKRP-related conditions.
Simone Thiele (Germany)
Simone Thiele is a member of the European Network TREAT-NMD as well as the German Network MD-NET and Curator of the FKRP registry for Germany. Besides, she is involved with the patient registries for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy which are organised by the Friedrich-Baur-Institute in Munich. Her other focus lies on the coordination of clinical trials.