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Showing 1–6 of 6 results
Advanced filters: Author: Anna M. G. Pasmooij Clear advanced filters

  • Induced pluripotent stem cells (iPSCs) have potential for regenerative medicine applications, but are generated with very low efficiency. Here, the authors show highly efficient reprogramming of human primary fibroblasts to iPSCs via the synergistic activity of synthetic modified mRNAs, mature miRNA mimics, and optimized culture methods.

    • Igor Kogut
    • Sandra M. McCarthy
    • Ganna Bilousova
    ResearchOpen Access
    Nature Communications
    Volume: 9, P: 1-15

  • Improved understanding of regulatory requirements by academic researchers can accelerate the translation of new medical interventions to the clinic. Here, we highlight the recommendations of the ‘Strengthening Training of Academia in Regulatory Science’ (STARS) project to improve the interaction and knowledge exchange between academics and regulators, and thereby advance academic drug development.

    • Viktoriia Starokozhko
    • Anne Heß
    • Anna M. G. Pasmooij
    Comments & Opinion
    Nature Reviews Drug Discovery
    Volume: 22, P: 251-252

  • A very small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them, known as N-of-1 therapies. This Review discusses advances and challenges for N-of-1 therapies based on cases in which they have been successfully developed, highlights why the traditional drug development and reimbursement pathway is not fit for purpose in this field, and provides a roadmap for the development of these individualized therapies.

    • Anneliene H. Jonker
    • Elena-Alexandra Tataru
    • Annemieke Aartsma-Rus
    Reviews
    Nature Reviews Drug Discovery
    Volume: 24, P: 40-56

  • The Remote Assessment of Disease and Relapse – Alzheimer’s Disease (RADAR-AD) consortium evaluated remote measurement technologies (RMTs) for assessing functional status in AD. The consortium engaged with the European Medicines Agency (EMA) to obtain feedback on identification of meaningful functional domains, selection of RMTs and clinical study design to assess the feasibility of using RMTs in AD clinical studies. We summarized the feedback and the lessons learned to guide future projects.

    • Gül Erdemli
    • Margarita Grammatikopoulou
    • Anna-Katharine Brem
    Comments & OpinionOpen Access
    npj Digital Medicine
    Volume: 7, P: 1-6