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Tag: rare diseases

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EMA launches consultation on proof-of-concept data in paediatric oncology

This initiative is also highly relevant from a rare diseases perspective, as paediatric cancers are rare conditions where small patient populations make robust non‑clinical proof‑of‑concept data essential for responsible and ethical trial initiation.
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Towards animal-free testing: virtual control groups gain regulatory acceptance

For rare diseases, where research often relies on limited animal models and small development teams, the regulatory acceptance of virtual control groups could help streamline early non‑clinical studies while maintaining a strong focus on patient safety.
Bojana Mirosavljevic

ERDERA interview with Bojana Mirosavljevic, rare disease patient advocate from Serbia and founder and president of Život/Life.

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Rare diseases clinical trials toolbox – public resources and main considerations to set up a clinical trial on medicinal products for humans in Europe

The Rare Diseases Clinical Trial Toolbox organizes and makes discoverable existing resources to support the design and conduct of clinical trials in rare diseases.
CELL & GENE THERAPY INSIGHTS

Breaking barriers in cell and gene therapy: advancing ATMPs through innovation and collaboration

Effective collaboration between industry, academia, and patient organizations is essential to advance standardized platform technologies. A recent example is the newly launched European Rare Diseases Research Alliance (ERDERA).

ERDERA's

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