February 28th is Rare Disease Day, a worldwide effort to call attention to the continuing need for research and awareness of diseases that affect a small percentage of the population. This year’s theme is “With research, possibilities are limitless.” As a science-focused company with a vision to improve human health by putting patients first, we are in complete agreement.
At Prothena, we apply our scientific expertise in protein misfolding toward the development of potential new therapies for two rare diseases known as AL amyloidosis and ATTR amyloidosis. Through this work, we have a firsthand appreciation about the dire need for greater education about rare diseases in the medical community and the general public.
While both AL and ATTR amyloidosis are rare, they represent the two most common forms of systemic amyloidosis. In patients with these diseases, toxic misfolded proteins accumulate and can cause dysfunction in the heart, kidneys, nervous system and other organs, often leading to life-threatening organ failure.
But because these diseases are rare, and because symptoms are so varied, systemic amyloidosis is frequently misdiagnosed. This means treatment is delayed, sometimes until it’s too late.
Amyloidosis patients are far from alone in this quandary. The National Institutes of Health counts 7,000 rare diseases; patients with any of these conditions are often unable to find answers and medical solutions they need. Awareness can help solve this.
Rare Disease Day is about making patients’ voices heard. Prothena honors this important mission and continues to work on behalf of patients with amyloidosis to advance potential new therapies. In recognition of Rare Disease Day, our employees helped raise awareness in South San Francisco with a march near our office in support of the many people worldwide suffering with rare diseases. We look forward to continuing our work in collaboration with patient communities and other life science companies who are committed to finding treatments for rare diseases.
Learn more about AL amyloidosis.