Tag: ATMPs (Advanced Therapy Medicinal Products)

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.
Pharmaceutical products

EU institutions agree next steps on reform of pharmaceutical legislation

Following the 11 December 2025 political agreement, EU institutions are moving towards formal adoption of a new Directive and Regulation that will reshape how medicines—including orphan and paediatric medicines—are developed, authorised and supplied across the European Union.
Advancement of Treatments for Rare Diseases

Postponed: Advancement of Treatments for Rare Diseases

The two-day event will focus on translating research and policy initiatives into tangible benefits for Rare Disease patients through better prevention, diagnosis, and treatment.
P065130-414294

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
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