SFL Pharma places prescription drugs onto the Swiss market, with a special focus on orphan drugs. Rare (or "orphan") diseases are life-threatening or chronically debilitating diseases that occur in less than 5 in 10,000 people in the Swiss community. There are approximately 7,000 rare diseases that may affect around 500,000 people in Switzerland.
Lysosomal Storage Disorders
Among orphan diseases, lysosomal storage disorders (LSD) pose a subgroup of 50 rare, medical conditions that affect approximately 10,000 people worldwide. LSDs are inherited metabolic disorders that are caused by a lack of specific enzymes that are responsible for the disposal of cellular substances in so-called lysosomes. As a result, these substances accumulate and cause toxicity in different organ systems such as kidney, muscle, lung, heart and brain. LSDs are severe, life-threatening diseases and put a heavy burden on patients, their caregivers and the healthcare system.