Rare disease can be complicated, and receiving a diagnosis is not always a straightforward process. If you or a loved one are undergoing diagnosis or treatment for a rare disease, you are not alone; and we recognise the importance of having accurate and reliable information to guide you through your treatment journey.
Unify Rare (an Alexion platform) is intended to educate and support patients by providing information about their rare disease. Please consult your doctor, nurse or pharmacist for further support.
Myasthenia gravis (MG) is caused by a disruption in the way the nerves and muscles communicate. It is characterised by varying muscle weakness and fatigue.1–3
NMOSD is a rare disorder characterised by unpredictable, intermittent episodes of vision impairment and mobility limitation.4,5 It is a consequence of the body mistakenly attacking healthy cells in the central nervous system (CNS).4,6
NF1 is a rare genetic condition that causes tumours (usually benign), known as neurofibromas, to grow along nerves.7,8 These can result in a range of symptoms, including brown patches on the skin, clusters of freckles and problems with bones, eyes and the nervous system.7 The condition progresses over time from childhood.8
aHUS is a rare and serious disease that affects both adults and children.9-11 aHUS is a condition caused when the complement system, which is part of the body’s immune system, becomes overactive.
Lysosomal acid lipase deficiency (LAL-D) is a rare, progressive and inherited condition caused by mutations in the LIPA gene which is responsible for the production of the lysosomal acid lipase (LAL) enzyme.12 LAL-D is characterised by problems with the breakdown and use of fats and cholesterol in the body.