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SUMMARY:Master Protocols for Rare Diseases
DESCRIPTION:NeuroNEXT 2025 Network-Linked Open Conference (NLOC)\nMaster Protocols for Rare Diseases: A Framework for Batten Disease \nThe Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) is hosting a virtual one-day virtual conference on clinical trials and clinical trial methods. This year’s NLOC puts Master Protocol trial design front and center — exploring how adaptive\, multi-arm frameworks can transform therapeutic development for Batten diseases\, other rare diseases\, and beyond. \nWhat to expect:\n🔹 Biostatistical insights on Master Protocols\n🔹 Platform trial case studies (ALS\, neurofibromatosis)\n🔹 Advocacy and patient perspectives\n🔹 Updates on the Batten disease therapeutic pipeline \n🔹 Workshops to develop umbrella and basket trial approaches for Batten diseases \nWho should attend?\nResearchers\, rare disease communities\, rare disease clinicians\, pediatric neurologists\, geneticists\, trial design experts\, biostatisticians\, trainees\, research coordinators\, project managers\, industry partners — and anyone invested in advancing therapy development for neurological conditions.
URL:https://ictr.johnshopkins.edu/event/master-protocols-for-rare-diseases/
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