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    Rare Disease

    University of Sheffield and Pfizer dispatch new consortium to quicken improvement of ATMPs

    The University of Sheffield and Pfizer have today (23 November 2020) established a new consortium which aims to standardize and accelerate the growth of Advanced Therapy Medicinal Products (ATMPs), allowing potentially transformative remedies to reach patients sooner. The new, five-year consortium, Accelerating Research and Innovation for Advanced Therapies (ARDAT), is supported by the Innovative Medicines

    Trinity crew uncovers mutations connected with early onset dementia

    Researchers at Trinity College Dublin today announced a significant advance in our knowledge of an early onset form of dementia which may also advance our understanding of conditions such as Alzheimer’s disease. Adult onset Leukoencephalopathy with axonal Spheroids and Pigmented glia (ALSP) is an ultra-rare condition characterized by mutations in a gene named Colony stimulating

    Analysts recognize interface among ALS and collection of DNA-RNA hybrids in the genome

    Researchers from the University of Seville and the University of Pavia have identified a connection between Amyotrophic Lateral Sclerosis (ALS) and the accumulation of DNA-RNA hybrids in the genome. The accumulation of these hybrids causes increased genomic damage and boosts genetic instability. This finding will make it possible to better understand the molecular basis of