Introduction to Translational Research for Rare Diseases 

Welcome and introduction

Setting the scene

What are the specific challenges and opportunities when developing medicines for rare diseases?

Developing a treatment for a rare disease: a case example

Key tools for rare disease research studies

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Welcome and introduction

How to plan the development of new medicines: an introduction

The target identification

Going from hits to leads

Drug repurposing

Developing therapies in rare diseases: the promises of new advanced therapies

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Welcome and introduction

What are animal models?

Challenges and new model options in rare diseases

Ethics and animal modeling

When to use which types of animal models?

Moving from preclinical to clinical studies

Why patient partnerships should be developed from the early stages of research

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Welcome and introduction

Avoiding common mistakes when moving from preclinical to clinical studies

From pre-clinical to first-in-human

A real planning process

Clinical trial design

Regulatory issues

Patient Reported Outcomes

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Welcome and introduction

Ending a clinical trial and drug approval process overview

Post marketing authorization studies

Preclinical & clinical data sharing and reuse

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