On Rare Disease Day, Helping Rare Shine Through
Rare Disease Day is observed every year on February 28 (or 29 in leap years)—the rarest day of the year.
Since its creation in 2008, Rare Disease Day has been uniting a diverse, global community around one purpose: To change and improve the lives of individuals living with a rare disease. A globally coordinated movement, Rare Disease Day provides a focal point and an energy around which members of the community, organizations and industry can rally to raise awareness and advocate for equity in social opportunity, health care, and access to diagnosis and therapies for people living with a rare disease.
The more people we can get to join in, the louder that voice is going to be, and better outcomes for patients and their families.
At Acadia, we are guided by our mission to advance breakthroughs in neuroscience to elevate life. Our dedication to the rare disease community is integral to fulfilling our purpose, and our commitment is rooted in insights from those living with and caring for people with rare diseases. By understanding their unique experiences, we can develop solutions that make a meaningful impact on their lives.
Here are just a few of the ways we are investing in the rare disease community:
- We launched a treatment option in the United States for Rett syndrome in adult and pediatric patients two years of age and older—and now, we are working to broaden access to this important therapy for families worldwide.
- Our growing rare disease pipeline includes treatments for three diseases that were previously untreatable and debilitating: In November 2023, we initiated a Pivotal Phase 3 trial of an investigational therapy for the treatment of hyperphagia in Prader-Willi syndrome, a rare genetic disorder that results in physical, mental and behavioral problems, a key feature being a constant sense of hunger that usually begins at about 2 years of age. We are also in the early development phases of an investigational candidate therapy for both Rett syndrome and Fragile X syndrome, an inherited genetic disorder that causes physical abnormalities, behavioral issues and a wide range of other health problems, including developmental delays and intellectual disabilities.
- We established the Rett Sibling Scholarship to shine a light on the often-unsung heroes of the Rett community, awarding 10 scholarships of $5,000 to eligible siblings of individuals living with Rett syndrome.
- We launched Rett Revealed to raise awareness of Rett syndrome and highlight the lives of Rett patients.
We also partner with local, national and international advocacy organizations to support community events and improve awareness and understanding of rare diseases.
Supporting the rare community allows me to raise my voice with thousands of other families advocating every day to bring more awareness to their daily journey.
Join us this Rare Disease Day in supporting the rare disease community through the National Organization of Rare Disorders (NORD) by showing your stripes.
On a rare day in February – and throughout the year – we can all contribute to making rare shine through.
