Observed annually at the end of February, World Rare (Orphan) Disease Day celebrates the global effort to raise awareness for rare diseases since its inception in 2008 by the European Organization for Rare Diseases.
The choice of February 29, a date known for its rarity, symbolizes the uncommon nature of these diseases, which are also known as orphan diseases. In non-leap years, the global community observes this day on February 28.
In our country, a disease is considered rare if it affects fewer than 10 people per 100,000 population. To date, there are approximately 8,000 known rare diseases.
Nanolek, a socially responsible pharmaceutical company, focuses on rare diseases. Its product portfolio includes an advanced treatment for Hunter syndrome, a rare disease.
Hunter syndrome, or mucopolysaccharidosis type II, is a hereditary, progressive disease that affects the skeletal, cardiovascular, and central nervous systems, as well as causing vision and ENT (ear, nose, and throat) problems.
Nanolek's drug is part of enzyme replacement therapy. At a recent roundtable discussion on mucopolysaccharidosis, ahead of World Rare Disease Day, healthcare professionals, advocates, and parents of affected children shared their experiences with the treatment. Currently, a new method for administering the drug is being introduced.
Healthcare experts emphasize the need not only to treat Hunter syndrome in children but also to monitor disease progression. Nanolek is committed to improving the lives of patients through innovative therapies.