Category: Data Hub

Advancement of Treatments for Rare Diseases

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.
Molecular therapy

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Outside traditional drug development pathways, clinicians and scientists face the challenge of systematically evaluating whether individual patients with severe ultrarare diseases might be eligible for and potentially benefit from individualized mutation-specific RNA therapies.
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
ERDERA JTC2026

ERDERA announces first Joint Transnational Call 2025 portfolio of preclinical therapy projects for rare diseases

Eighteen international pre clinical therapy projects selected under ERDERA’s first Joint Transnational Call will accelerate treatment options for people living with rare diseases across Europe and beyond
HLM Rare Diseases Brussels 2025

HLM Rare 2025 in Brussels: forging a European Declaration for rare and complex diseases

Three days in Brussels help advance the European Declaration on Rare and Complex Diseases
CRN Conference Group Photo

Clinical experts and patient advocates gather in Heidelberg to turn discussion into action on global rare disease research

From shared standards to local impact, the 2nd Clinical Research Networks Conference agreed concrete next steps for interoperable clinical research networks worldwide
ERDERA JTC2026

ERDERA launches 2026 Joint Transnational Call on diagnostics for unsolved rare diseases

Call now open; preproposals due 12 February 2026
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.
European Medicines Agency (EMA)

EMA public consultation on phage therapy medicinal products

EMA invites comments on new quality guideline for bacteriophage‑based medicines to support safe phage therapy development in Europe.
orphanet

The involvement of underrepresented countries in the European rare diseases research alliance: Morocco’s assets

Using Morocco as a case study, this paper shows how underrepresented countries contribute unique genetic insights to rare disease research and why their integration into initiatives like ERDREA is essential for both scientific progress and equity.
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