Category: Clinical Research

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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.
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.
15th Balkan Congress

A landmark gathering brings together regional leaders in human genetics to reshape genomic medicine

From 9 to 11 October in Bled, the 15th Balkan Congress and 3rd Alpe Adria Meeting put rare disease diagnostics and predictive genomics centre stage.
SANT Launches Public Consultation on Rare Diseases

EU consultation on disability rights seeks evidence to strengthen inclusive research and data ecosystems

Inviting views on how a stronger EU disability strategy up to 2030 can support inclusive research, data sharing and innovation.
are Diseases in a Changing & Competitive Europe: Shaping policies to address the unmet needs of people living with rare diseases

13th European Conference on Rare Disease and Orphan Drugs

The European Conference on Rare Diseases & Orphan Products (ECRD) is the largest patient-led policy-shaping event on rare diseases in Europe.
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.
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