The Longest Journey: How Rare Disease Networks are Transforming Patient Journeys
Rare Disease Networks Perspectives Series – Blog 4/5 The Longest Journey: How Rare Disease Networks are Transforming Patient Journeys There is a particular kind of
Rare Disease Networks Perspectives Series – Blog 4/5 The Longest Journey: How Rare Disease Networks are Transforming Patient Journeys There is a particular kind of
Regulation, by its nature, is cautious. Agencies tasked with protecting public health are not designed to move fast; they are designed to be rigorous. Yet
For most of the twentieth century, the pharmaceutical industry’s relationship with rare diseases could be summarised in a single phrase: studied indifference. The economics were
Across the world, an estimated 300 to 400 million people live with a rare disease. Yet for most of the twentieth century, these individuals lived
The Day Something Felt “Not Quite Right” One ordinary day, I suddenly had a nosebleed. That alone isn’t unusual—I’ve had them before. But this time,
By Leigh Hart, Senior Moderator @ LDA Research Rare diseases affect an estimated 300–400 million people worldwide, yet progress in diagnosis and treatment has historically
At LDA Research, we know that behind every data point is a real person, a real family, and a real story. We’re sharing one of
If 2024 was the year medicine proved that steady, methodical progress could deliver profound impact without spectacle, 2025 is the year those advances began to
In rare diseases, patients aren’t on the sidelines – they are central partners. Their lived experience fills critical evidence gaps and shapes what “meaningful outcomes”
At LDA Research, we know that behind every data point is a real person, a real family, and a real story. We’re sharing one of
Senior moderator Leigh Hart explores the role of pharmaceutical companies in the Rare Disease Networks, what was planned and how it has worked out. Rare
Erin Paterson shares her experience of being gene positive for the rare Huntington disease, and how she is on a mission to positively impact other