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at Sapienza University of Rome. This PhD project focuses on the preclinical evaluation of pharmacological therapies for megalencephalic leukoencephalopathy with subcortical cysts (MLC), a rare leukodystrophy
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through to preclinical characterisation. Your work will include: Selecting GPRC5B-targeting nanobodies from immune and synthetic nanobody libraries and evaluating AI-generated candidates. Characterising
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project focuses on characterising leukodystrophy mouse models and evaluating promising proof-of-concept interventions. You will conduct longitudinal studies in mouse models, with a particular focus on
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will contribute to the development and evaluation of gene therapy approaches for PMLD1. Your work includes: establishing and using isogenic iPSC-derived myelinating organoids as a human in vitro disease
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fragment-based drug discovery to identify and characterise promising compounds. Contributing to the development of a functional GPCR assay pipeline and the evaluation of lead molecules in collaboration with
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is the Medicines Evaluation Board (MEB) in Utrecht, the Netherlands. You will be employed by the MEB and embedded in the POLARIS consortium. You will be supervised by Dr Marjon Pasmooij and work
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you will contribute to evaluating access pathways for advanced therapy medicinal products (ATMPs). Your academic embedding will be at the Amsterdam Leukodystrophy Center, with the anticipated PhD
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the Graduate School of the Cyprus Institute of Neurology and Genetics. You will pursue a PhD degree in Neuroscience. You will contribute to the development and evaluation of gene therapy approaches for PMLD1
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understanding of what matters to patients and families throughout the course of the disease. The project has three connected research areas: Quality of life: evaluating existing quality-of-life measures and
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, disease monitoring and treatment evaluation. You will work closely with researchers in human genetics, computational biology and clinical neurology and collaborate with POLARIS partners across Europe