Tag: Gene Therapy

the power behind erdera2 (1)

Undiagnosed Day 2026 | Where Diagnosis Becomes Hope: The Power of Collaboration and Technology in Rare Diseases

This meeting will focus on practical approaches to phenotyping and diagnosis in undiagnosed conditions, including how to define next steps when a diagnosis remains uncertain, and how to strengthen pathways and collaboration around undiagnosed care.
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.
man working in a lab

ERDERA pre announces 2026 Call for Proposals on resolving unsolved rare diseases

The 2026 Joint Transnational Call will be launched on 10 December, with a free information webinar on 16 December
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ERDERA and the European Genomic Data Infrastructure project work together to strengthen rare disease data, diagnostics and research

Fruitful collaboration ahead
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

15th Balkan congress of human genetics and 3rd Alpe Adria meeting of human genetics

15th Balkan congress of human genetics and 3rd Alpe Adria meeting of human genetics

The 15th Balkan Congress of Human Genetics and the 3rd Alpe Adria Meeting of Human Genetics will take place from 9–11 October 2025 at the Rikli Balance Hotel, Bled, Slovenia.
Exterior of European Medicines Agency in Amsterdam

EMA warns of unregulated advanced therapies in the EU

ERDERA's

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