![An Alexion employee working in a lab](/-/media/alexion_comredesign/images/non-patient-images/lab-01-s.jpg?rev=0be9b4d9451e4defb11e23a7e6d49d49?mw=375 375w,
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CONDITIONS WE TREAT
Understanding Rare Diseases, Every Day
We’re dedicated to delivering life-changing medicines for those with rare and devastating diseases. Our innovation begins when we understand the challenges that people living with these rare diseases and their families face every day.
![Julia, diagnosed with aHUS at 15 years old, riding bikes with her sister](/-/media/alexion_comredesign/images/realstories/ahus/julia/julia-02-r.jpg?rev=4aa6c1dc7f634afeaaae2999328d679e?mw=375 375w,
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aHUS
Atypical Hemolytic Uremic Syndrome
aHUS is an ultra-rare genetic, chronic, potentially life-threatening disease that can progressively damage vital organs, such as the kidneys.1
LEARN MORE![Aimee, diagnosed with gMG](/-/media/alexion_comredesign/images/realstories/gmg/aimee/aimee_07321_513x320_real-patients-and-stories.jpg?rev=612226c326ac4a0b99e9e26611be4c41?mw=375 375w,
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gMG
Generalized Myasthenia Gravis
gMG is a debilitating, chronic and progressive autoimmune neuromuscular disease that can occur at any age but most commonly begins for women before the age of 40 and men after the age of 60.2-5
LEARN MORE![Tanner, diagnosed with HPP at 4 days old, with a stuffed animal](/-/media/alexion_comredesign/images/realstories/hpp/tanner/tanner-01-r.jpg?rev=ba4b77113f584753979533caaffd2564?mw=375 375w,
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HPP
Hypophosphatasia
HPP is a genetic, chronic, progressive, and life-threatening metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications.6
LEARN MORE![Albie, diagnosed with LAL-D as an infant, holding a puppy](/-/media/alexion_comredesign/images/realstories/lal-d/albie/albie-04-r.jpg?rev=5c6742cf26fa4bfe8a8ca8df716d6fcd?mw=375 375w,
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LAL-D
Lysosomal Acid Lipase Deficiency
LAL-D is a genetic, chronic and progressive ultra-rare metabolic disease in which uncontrolled accumulation of cholesteryl esters and triglycerides may lead to multi-organ damage in infant, pediatric, and adult patients and premature death in infants.7
LEARN MORE![](/-/media/alexion_comredesign/images/conditions/fx-bleed/bleed-r.jpg?rev=e4cbf17705534d62a25f1181bea0983c?mw=375 375w,
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NF1 PN
Neurofibromatosis Type 1 Plexiform Neurofibromas
NF1 is a rare, progressive, genetic condition impacting multiple body systems characterized by benign tumors called plexiform neurofibromas (PN) that develop along nerve sheaths throughout the body.8-11 Depending on their size and location, PN can cause a range of clinical issues.12,13
LEARN MORE![KIM](/-/media/alexion_comredesign/images/realstories/nmosd/slide-5_kim_2560x1600.jpg?rev=2e422a458bd041848504d18372f7915a?mw=375 375w,
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NMOSD
Neuromyelitis Optica Spectrum Disorder
NMOSD is a rare and devastating complement-mediated disorder of the central nervous system (CNS) characterized by relapses where each individual attack results in cumulative disability.14-18
LEARN MORE![Joe, diagnosed with PNH at 24 years old, training for mixed martial arts](/-/media/alexion_comredesign/images/realstories/pnh/joe/joe-02-r.jpg?rev=402818e3079e4ca59550b5c6222b7971?mw=375 375w,
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PNH
Paroxysmal Nocturnal Hemoglobinuria
PNH is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells).13,14
LEARN MOREReferences:
- Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med. 2009;361(17):1676-1687.
- Huda R, Tüzün E, Christadoss P. Targeting complement system to treat myasthenia gravis. Rev Neurosci. 2014;25(4):575-583.
- Howard JF, Barohn RJ, Cutter GR, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized Myasthenia Gravis. Muscle Nerve. 2013;48(1):76-84.
- National Institute of Neurological Disorders and Stroke. Myasthenia Gravis Fact Sheet. http://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm. Accessed October 22, 2019.
- Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009;8(5):475-490.
- Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10(suppl 2):380-388.
- Kanuma® [package insert]. New Haven, CT: Alexion Pharmaceuticals, Inc; 2015.
- Tonsgard JH. Clinical manifestations and management of neurofibromatosis type 1. Semin Pediatr Neurol. 2006;13(1):2-7.
- Hirbe AC, Gutmann DH. Neurofibromatosis type 1: A multidisciplinary approach to care. Lancet Neurol. 2014;13(8):834-843.
- Blakeley JO, Plotkin SR. Therapeutic advances for the tumors associated with neurofibromatosis type 1, type 2, and schwannomatosis. Neuro Oncol. 2016;18(5):624-638.
- Dombi E, Ardern-Holmes SL, Babovic-Vuksanovic D, et al. Recommendations for imaging tumor response in neurofibromatosis clinical trials. Neurology. 2013;81(21 Suppl 1):S33-S40.
- Korf BR, Rubenstein AE. Neurofibromatosis: A Handbook for Patients, Families, and Health Care Professionals. New York, NY: Thieme Medical Publishers; 2005.
- Hersh JH. Health supervision for children with neurofibromatosis. Pediatrics. 2008;121(3):633-642.
- Wingerchuk DM. Neuromyelitis optica spectrum disorders: critical role of complement-dependent cytotoxicity. Neurology Reviews. 2017;(suppl):S1-S4.
- Mealy MA, Boscoe A, Caro J, Levy M. Assessment of patients with neuromyelitis optica spectrum disorder using the EQ-5D. Int J MS Care. 2019;21(3):129-134.
- Jarius S, Ruprecht K, Wildemann B, et al. Contrasting disease patterns in seropositive and seronegative neuromyelitis optica: a multicentre study of 175 patients. J Neuroinflammation. 2012;9:14.
- Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the United Kingdom and Japan. Brain. 2012;135(pt 6):1834-1849.
- Jiao Y, Fryer JP, Lennon VA, et al. Updated estimate of AQP4-IgG serostatus and disability outcome in neuromyelitis optica. Neurology. 2013;81(14):1197-1204.
- Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333(19):1253-1258.
- Kelly R, Richards S, Hillmen P, Hill A. The pathophysiology of paroxysmal nocturnal hemoglobinuria and treatment with eculizumab. Ther Clin Risk Manag. 2009;5:911-921.
US/ALL-A/0042
![The Trendys, diagnosed at 17, 14, 11, and 9 with LAL-D](/-/media/alexion_comredesign/images/realstories/lal-d/the-trendys/trendy-02-f.jpg?rev=95ba41018f7a48858288eb817d1e0e0a?mw=375 375w,
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Now we knew what we were up against, we knew we could fight.”REBECCA, MOM OF THE TRENDY BROTHERS LIVING WITH LAL-D