Rett syndrome (RTT) is a genetic disease that is caused by abnormalities in a gene called MECP2 which is important for the normal functioning of nerve cells. It is a rare disease which almost always affects females. Females with RTT lose their ability to properly control their muscles, have feeding difficulties and learning disabilities. Other symptoms include difficulty breathing, irregular heartbeat, sleeping problems, constipation, repetitive hand movements and fits.
Sarizotan is an oral medicinal product that is being developed for the treatment of respiratory symptoms associated with Rett syndrome. Sarizotan works by binding to serotonin and dopamine receptors. By stimulating serotonin and dopamine receptors, sarizotan replaces the effect of some of the missing serotonin in the brain and spinal cord. This is expected to help restore normal breathing rhythm in patients with Rett syndrome. Currently, there is no cure for Rett syndrome and treatment focuses on managing the symptoms. If licensed, sarizotan could become the first therapy approved for treatment of Rett Syndrome patients.
BIIB092 is a product that is being investigated for the treatment of progressive supranuclear palsy (PSP). PSP is a rare condition that is a result of destruction of nerve cells in certain parts of the brain causing problems with balance, movement, vision, speech and swallowing. In patients with PSP, an abnormal form of a protein called tau accumulates in specific areas of the brain by spreading from brain cell to brain cell leading to their damage. Over time, PSP gets progressively worse, with people becoming severely disabled within three to five years of onset. Currently, there is no cure for PSP and no treatment to slow down the disease.