Category: Expertise Services

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New survey launched to improve rare diseases clinical trial enrolment

The RealiseD project launched a new multi-stakeholder survey that invites the rare disease community to share insights and help improve clinical trial enrolment
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.
RealiseD webinars

RealiseD webinar series: Helping re‑shape rare disease clinical trials

13 January – 10 February 2026 | Online webinar series to advance best practices in clinical trial design for rare and ultra rare diseases.
Non-human primate hand via pickpik.com (CC0)

EMA public consultation on non‑human primate use in safety testing

EMA seeks stakeholder input on new reflection paper to reduce and replace non‑human primate use in medicines safety testing through 3Rs approaches.
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.
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
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.
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.
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.
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