Tag: ATMPs (Advanced Therapy Medicinal Products)

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Joining the Dots: EURORDIS’ role in ERDERA and the reshaping of rare disease research

EURORDIS champions patient-centred innovation in rare disease research
European Medicines Agency (EMA)

First Italian-made gene therapy for Wiskott-Aldrich Syndrome

On 13 November in Milan, EMA’s expert committee backs Italy’s first home‑grown gene therapy for people in Europe living with Wiskott–Aldrich Syndrome.
World Orphan Drug Congress USA 2026

World Orphan Drug Congress USA 2026

More than 280 speakers from industry, regulators, patient organisations, investors and healthcare providers, and aims to showcase new science, technologies and policies that can accelerate orphan drug development and improve access to therapies for people living with rare conditions.
High-Level Meeting on European Research and Innovation for Rare Diseases

High-Level Meeting on a European Research and Innovation Ecosystem for Rare Diseases

Gathering key stakeholders, including EU policymakers, industry leaders, patient advocacy groups, researchers, and healthcare providers. Its goal is to identify concrete actions and incentives to enhance innovation across the rare disease ecosystem.
Triplet repeat expansion project sets benchmark for public-private collaboration

A first in personalised medicine: CRISPR treatment for a baby sparks new debate on rare disease therapies

ERDERA group photo from the workshop

Multi stakeholder workshop advances prioritisation of rare diseases for ATMP development

A practical framework in the making
EU rare disease conference concludes in Warsaw with renewed focus on early diagnosis and collaborative research

EU rare disease conference concludes in Warsaw with renewed focus on early diagnosis and collaborative research

Representatives from patient organisations advocated for robust data platforms and research partnerships
Rare Disease conference kicks off in Warsaw

Rare Disease conference kicks off in Warsaw: Building momentum for a unified EU strategy

Rare disease experts to gather in Warsaw to shape Europe’s future strategy

Rare disease experts to gather in Warsaw to shape Europe’s future strategy

Taking place on 10 April 2025 at the Medical University of Warsaw, Poland
Exterior of European Medicines Agency in Amsterdam

EMA warns of unregulated advanced therapies in the EU

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