Pathologies Orphelines

REW Keynote: Gene Therapy and Gene Editing for Orphan Diseases

Children's National Hospital

Emerging Therapies for Orphan Diseases: Ethics and Economics (English Version)

McGill University

MOPD 2023 - Rare and Orphan Diseases Session

LiVDerm: Revolutionizing Dermatology Education

AKU The Forgotten Orphan

Model Research: The Rare and Orphan Disease Center at The Jackson Laboratory

The Jackson Laboratory

VP of C-Path Rare and Orphan Disease Programs Collin Hovinga Talks Rare Disease Drug Development

Critical Path Institute

Addressing Orphan and Rare Diseases

InsideScientific

The Manton Center and Orphan Disease: Overview

Boston Children's Hospital

Patient and Carer Perspectives and Real-World Evidence in Orphan Disease HTA Submissions

Defining Rare Diseases and Orphan Drug Act

The Manton Center for Orphan Disease: Research

Boston Children's Hospital

How to Simplify Genetics & Raise Awareness Around Orphan Diseases

Impetus Digital

Galactosemia - A family's fight against this rare orphan disease

Boston Children's Hospital

High Cost Orphan Disease Drugs Explained

AHealthcareZ - Healthcare Finance Explained

The Manton Center for Orphan Disease: Purpose and Mission

Boston Children's Hospital

The Manton Center for Orphan Disease: Future Plans

Boston Children's Hospital

Maladies orphelines : un film pour sensibiliser au Syndrome de Lafora

Patriotes pour l'Europe - France 🇫🇷

Advances in treatments for orphan diseases

Chronicle 5 WCVB

Biological sibling turns her down because of schizophrenia #shorts

Best Documentary

Piloting Pathways for “Interventional Genomics” in Orphan Neurologic Disease - CIM