Fiona Brock MSc CStat

If you want to help create a future where: 1️⃣ people with rare diseases have faster diagnoses and better treatment options, and 2️⃣ innovators find a coordinated, sector-wide approach to get new tests and treatments to market, and help them stay there ➡️ read our latest report with Genetic Alliance UK: https://lnkd.in/e8iPr7Xi It recommends 10 actions that government, regulators, industry, academia, funders, clinicians, and patient groups can take to #ChangeTheRareFuture for those living with rare conditions.

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We are delighted to join the World Health Organization's (WHO) new Global Clinical Trials Forum which officially launched earlier today. The forum is designed to strengthen clinical trial environments and infrastructure across the world. It will support the implementation of the WHO's Guidance for Best Practices for Clinical Trials, which saw the UK recognised as a country with a strong clinical trial infrastructure. Read more about the news by following the link in the comments below. #ClinicalTrials #Research #NHS #WHO #GlobalClinicalTrialsForum

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ISPOR announced details for its #ISPOREurope 2025. The conference is the leading global conference in Europe for health economics and outcomes research (HEOR) and is scheduled for 9-12 November in Glasglow, Scotland, UK with the theme, “Powering Value and Access Through Patient-Centered Collaboration.” #ISPORNews #HEOR Learn more: https://ow.ly/jgFA50X9ZHQ

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With the National Cancer Plan expected in the coming months, its contents sparked discussions on the sidelines of the Labour Party Conference.   John McNeill, Oncology Business Unit Director at MSD in the UK, explores in The House Magazine how the Plan can truly improve care and outcomes for cancer patients across England.   From reducing disparity in care to preparing for the next generation of treatments, the National Cancer Plan presents a rare opportunity to address the challenges facing cancer patients in England today and enable the NHS for tomorrow.   See below three elements we believe the Plan should deliver on👇and read the full article here 👉 http://bit.ly/4ogj63z   GB-NON-11930

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'We need to move with the times' - Matthew Durdy, Chief Executive, Cell and Gene Therapy Catapult.   In this Pink Sheet interview, Matthew Durdy, CGT Catapult, discusses how recent changes in UK drug regulations could impact the #cell and #gene therapy industry, and how the Medicines and Healthcare products Regulatory Agency (MHRA)'s new reforms in decentralised manufacturing are a positive signal for the UK's #CellTherapy ecosystem.   Read the interview: https://lnkd.in/eK4AR8BG   #ATMPs #AdvancedTherapies Citeline

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Today we’re pleased to share our Clinical Academics Summit statement, signed by over 40 organisations 🤝 An alarming decline in clinical academic posts threatens the UK’s ability to develop new treatments and remain a global leader in health research ⚠️ Clinical academics are vital in connecting research and patient care – shaping research priorities through frontline experience and bringing new scientific discoveries straight to patients 🧑‍⚕️ ✨ Earlier this year, we convened leaders from universities, research funders, industry partners, the NHS and Government at a summit to commit to action to help reverse this trend. The statement of ambition and intent seeks to address the OSCHR reports’ recommendation to increase the number of permanent clinical academic posts, year-on-year. Our summit attendees committed to five priorities: 1️⃣ Raise awareness of the benefits and opportunities in clinical academia 2️⃣ Prioritise funding for new posts 3️⃣ Champion clinical research as central to care delivery 4️⃣ Build local partnerships across academia, industry, funders and the NHS 5️⃣ Tackle barriers faced by underrepresented groups including nurses, midwives, allied health professionals, ethnic minorities and women 📖 Read the full statement: https://lnkd.in/eU4PiVbS #ClinicalAcademics #HealthResearch #MedicalResearch

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Working backwards from a clinical effect might seem an odd approach, but in the case of the deubiquitinase Ubiquitin Specific Protease USP30, this has been the challenge for Darragh O’Brien’s research. USP30 inhibitors had recently advanced to clinic trials, but the mechanism of action (MoA) was still not yet fully established.   At the outset of the study, structural information for USP30 was not available, making it difficult to look at the conformation of the USP30 inhibitor binding pocket. Darragh used a structural proteomics approach to characterise the binding site of different types of USP30 inhibitors on the protein, as traditional crystallography had not yet been successful. Activity-Based Protein Profiling Mass Spectrometry (ABPP-MS), Hydrogen-Deuterium eXchange Mass Spectrometry (HDX-MS) and other supporting biophysical techniques coupled with enzyme kinetics enabled the development of a structural model which showcased the potency, activity, and selectivity of different USP30 inhibitor classes.   The data obtained from the highly integrative approach resulted in a proposed MoA for non-covalent USP30 inhibition. The work has since been extended to include covalent inhibitors, and has been confirmed by others in the field, paving the way for the development of next-generation inhibitors of USP30 targeting neurodegenerative disease and mitophagy-related disorders.   Darragh O'Brien is a Researcher in Neurodegenerative Disease and Head of Structural and Mechanistic Proteomics at the Centre for Medicines Discovery, University of Oxford. His talk ‘Targeting mitochondrial dysfunction in neurodegenerative disease - The advent of potent and selective USP30 inhibitors’ will be presented on Wednesday 22nd October at ELRIG’s Drug Discovery 2025, in the scientific track ‘From bench to brain: Hit-finding approaches in neurodegenerative disease therapeutics in collaboration with Alzheimer’s Research UK’. Register now to hear his presentation: https://lnkd.in/e8c59Ung  #USP30 #ABPP #HDX  #deubiquitinylases #Parkinsonsdisease #mitophagy #drugdiscovery #SAR  #moleculardocking #structuralproteomics

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One in two of us will be affected by dementia in our lifetime. The team at Cambridge’s ALBORADA Drug Discovery Institute, funded by Alzheimer's Research UK, is dedicated to finding better treatments so people can live well and stay independent in later life. Animal research is vital. Before any new drug can go into human clinical trials, it must be safety-tested as thoroughly as possible. A mouse model allows the team to test potential new drug molecules – and see which act best on reversing damage to the brain. They need to test on whole brains because they’re complex, and they also need to understand how the whole body is affected by the drug to work out the right dosage and look for side-effects. Learn more about how mice are crucial in dementia research 👉 https://bit.ly/3H4OaTV #Dementia #AnimalResearch #CambridgeUniversity

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