February 28th is Rare Disease Day, organized by the global rare disease advocacy community to raise awareness for rare diseases and their impact on patients, caregivers and families. Mallinckrodt Pharmaceuticals recognizes the importance of organizations working together and is proud to support the U.S. sponsor of Rare Disease Day—the National Organization for Rare Disorders (NORD)—and other advocacy groups in these efforts.
More than 7,000 rare diseases impact 25 to 30 million Americans.[i] Half of those affected by a rare disease are children.[ii] While scientific advances have helped to identify more rare diseases, why and how they develop, as well as potential therapies, 95 percent of rare diseases today are still without a treatment.2 Moreover, symptoms of rare diseases aren’t often immediately recognized or they’re mistaken for other conditions. Patients can spend months or years getting a diagnosis, further delaying treatment. Once a diagnosis is made, resources and knowledge to help patients stay informed about the latest research and advances can be difficult to find.
Patients with rare diseases and their families navigate many unknowns and face daily challenges and difficulties. Their medical care typically requires unique, patient-centered approaches that address their special needs and enhance their quality of life.
As advocates and patient champions, our team strives to help meet the needs of underserved patients along every step of their journey through research, education and outreach. We connect patients and care providers, and support trial design and recruitment. Partnerships with advocacy organizations like NORD are another important way to address gaps in knowledge so that patients, families, caregivers and providers can better understand how to safely and effectively manage a chronic serious illness.
As a company that aims to solve complex challenges with innovative solutions, Mallinckrodt is committed to advancing knowledge and understanding to help the community achieve the best possible outcomes.
On Rare Disease Day, we honor the patients who inspire us every day. For more information, visit www.rarediseaseday.org.
[i] National Institutes of Health, Genetic and Rare Diseases Information Center. FAQs About Rare Diseases. Available at https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases. Accessed February 7, 2019.