Alexion Pharmaceuticals (Nasdaq:ALXN) joins the European Organization for Rare Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and patient organizations worldwide in recognizing Rare Disease Day 2014, a global effort to increase awareness of rare diseases, their profound impact on patients and the need for improved diagnosis and treatment.
Alexion supports key objectives of EURORDIS, NORD and other patient organizations through its mission to develop and deliver life-transforming therapies for patients worldwide who suffer from severe and life-threatening rare diseases.
Diagnosis and Treatment Challenges for Rare and Ultra-Rare Diseases
Many rare and ultra-rare diseases are chronic, progressive and marked by continuing pain, severe disability and high mortality. Few physicians are familiar with diagnosing and treating these illnesses, which frequently leads to missed, delayed or inaccurate diagnoses even when an approved, effective therapy is available. 1 According to global survey results published in the current issue of The Journal of Rare Disorders (JRD), conducted on behalf of Global Genes, patients living with rare diseases visited an average of 7.3 physicians before receiving an accurate diagnosis. 2 The survey also reveals that 44 percent of patients surveyed believed that a slow diagnosis resulted in delayed treatment with a negative impact on their condition. 2“Patients with rare and ultra-rare life-threatening disorders often face long delays in receiving an accurate diagnosis. Without an accurate diagnosis, or access to effective treatment options, patients too often are at risk for catastrophic medical consequences,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “On this Rare Disease Day and every day, through our continued commitment to breakthrough medical research, each of us at Alexion are focused on improving the knowledge of rare diseases and developing and delivering therapies to transform the lives of these patients worldwide.” Delivering Life-Transforming Therapies Across the Globe Alexion’s research and development programs are focused on highly innovative therapies with the potential to transform the lives of patients with severe and life-threatening ultra-rare disorders and for which there are no effective treatment options. The company’s development programs are solely focused on:
- Severe disorders with devastating and life-threatening medical consequences
- Disorders with ineffective, or no treatment options
- Disorders that are ultra-rare and affect very small numbers of patients