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HEДокторантска мрежа2026–2030

POLARIS · Progress in Leukodystrophies: Advance, Rate and Implement new treatment Strategies

„Хоризонт Европа“ — Действия „Мария Склодовска-Кюри“

Период
2026-10-01 → 2030-09-30
Финансиране от ЕС
4 624 038 €
Участници
32
Схема
HORIZON-TMA-MSCA-DN

Линиите свързват координатора с партньорите. За проекти отпреди 2014 г. CORDIS не винаги дава точни координати. Тези точки са на ниво град или държава.

Накратко на български

Левкодистрофите са тежки генетични заболявания на бялото вещество в мозъка, за които се разработват нови генни и лекарствени терапии. Тези проучвания помагат за създаването на по-достъпни методи за лечение и по-добри инструменти за оценка на състоянието на пациентите.

Този кратък обзор е генериран от изкуствен интелект

Кратко обяснение, генерирано от езиков модел по текста на CORDIS. Оригиналът е по-долу.

Цел на проекта

Leukodystrophies are severe genetic disorders of the brain white matter that cause profound disability and premature death, mostly in children, and lack effective treatments. POLARIS aims to accelerate therapy development by training the next generation of leukodystrophy scientists capable of collaborating across the full spectrum of discovery, development, evaluation and implementation of innovative and accessible therapies. Researchers will develop gene-therapy approaches (RO1), pharmacological strategies (RO2) and treatment-evaluation tools (RO3) to advance therapies for leukodystrophies and strengthen R&I frameworks for other rare diseases.A key barrier to progress is fragmentation: discovery, preclinical development, outcome definition and regulatory pathways rarely coexist in a single environment. POLARIS bridges this through 3 cross-cutting themes: (I) Affordable, Accessible and Timely—embedding cost, accessibility and real-world feasibility from the earliest stages; (II) Relevant Outcomes—focusing on what matters to patients, caregivers, clinicians, regulators and society; and (III) Transferable Knowledge & Tools—producing broadly applicable insights and platforms beyond individual diseases. The programme integrates all relevant stakeholders, including strong engagement of patient organisations, industry and public bodies.POLARIS generates major scientific advances through innovative gene-therapy and pharmacological platforms, validated biomarkers and harmonised outcome measures that directly support future clinical trials. Its end-to-end translational pipeline creates scalable technologies relevant to a wide range of rare neurological disorders. POLARIS enhances researchers’ career prospects, skills, employability and contributions to Europe’s innovation capacity. By reducing development barriers and enabling more effective, affordable treatments, POLARIS delivers substantial economic and societal benefits for patients, families and healthcare systems.

Оригинален текст от CORDIS (на английски).

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Данни: CORDIS, © Европейски съюз