TURNING THE TIDE FOR RARE DISEASE

https://rarerevolutionmagazine.com/category/rare-insights/turning-the-tide-for-rare-disease/ Michèle Melhem, head of rare disease, International Center of Excellence at Biogen discusses the need for urgent and authentic multi-stakeholder collaboration to co-create solutions that could accelerate rare disease innovation, underscoring Biogen’s commitment to true patient-centricity Every day, families living with rare diseases face a reality few can imagine; years of uncertainty, unanswered questions and decisions made without clear guidance. For many, the journey begins with a long wait, with research showing that an accurate diagnosis takes an average of 4-8 years.1 Parents watch key childhood milestones slip by while searching for answers that feel just out of reach. Even when a diagnosis comes, hope is often tempered by the fact that most rare conditions still have no approved treatment.