Switzerland

A rare, chronic, progressive and potentially life-threatening blood disorder. It is characterised by red blood cell destruction within blood vessels (also known as intravascular haemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots).

A rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.

A rare, progressive, genetic condition characterised by non-malignant (non-cancerous) tumors called plexiform neurofibromas (PN) that develop in the brain, and along the spinal cord and nerve.

A rare, autoimmune neuromuscular disease characterised by loss of muscle function and severe muscle weakness.

A rare autoimmune disease of the central nervous system, characterised by unpredictable relapses which can result in cumulative disability.

A rare, genetic (inherited), metabolic disease characterised by impaired mineralisation (“calcification”), the process that hardens and strengthens bones and teeth. This can lead to poor growth and development, weakness and deformity of bones and other skeletal abnormalities, and premature loss of teeth with the root intact.

A genetic, and progressive ultra-rare metabolic disease associated with multiorgan damage in infant, paediatric and adult patients, and premature death in infants.