Rare Disease
Expertise: Rare Disease 73 results
In recent years, the pharmaceutical landscape for rare diseases has witnessed a remarkable evolution. Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than 300 million people worldwidei and account for a significant portion of the global clinical development pipeline. This blog delves into the rise of…
Rare Disease
World Symposium (Lysosomal Storage Disorder)
Rare Disease
WODC US
In rare diseases, patient opinion and insight often go unheard. However, for sponsors of rare disease clinical trials, study success often rests on incorporating the patient perspective as early as possible in development. Many patients with rare diseases have undertaken a long journey to diagnosis, so participating in a study may feel like a huge…
Rare Disease