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When Albie was born, his mom, Charlotte, said that he was “absolutely fine. Everything was fine for about two weeks.”
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As a father of two young children, Donnan recalls being afraid of what the future would hold prior to his diagnosis.
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Married with a newborn son, Ruthie was scared to learn that PNH was a life-threatening illness that at the time had no approved treatment.
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At only three months old, Evie's parents were told that her case was severe and that there were no approved or effective treatment options.
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Although physical challenges required Liz, her husband, and their children to adjust their lives in many ways, she doesn't let her disease hold her back.
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Tristan's parents experienced a whirlwind of emotions and fear after learning their 5-year-old son had such a rare and serious disease.

CONDITIONS WE TREAT

Fighting gMG Every Day

What is generalized Myasthenia Gravis (gMG)?

Myasthenia gravis (MG) 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.^1-4 It typically begins with weakness in the muscles that control the movements of the eyes and eyelids, and often progresses to the more severe and generalized form, known as gMG, with weakness of the head, neck, trunk, limb, and respiratory muscles.⁴

Aimee, diagnosed with gMG

AIMEE
LIVING WITH gMG

Patients with gMG can suffer from slurred speech, choking, impaired swallowing, double or blurred vision, disabling fatigue, immobility requiring assistance, shortness of breath, and episodes of respiratory failure. Complications, exacerbations, and myasthenic crises can require hospital and intensive care unit admissions with prolonged stays and can be life-threatening.^2,3,5 While most gMG symptoms can be managed with existing MG therapies, 10% to 15% of patients fail to respond adequately to or cannot tolerate multiple therapies for MG and continue to suffer profound muscle weakness and severe disease symptoms that limit function.^6-8

In patients with anti-acetylcholine receptor (AChR) antibody-positive MG, the body’s own immune system turns on itself to produce antibodies against AChR, a receptor that plays an important role in the communication between nerves and muscles.^2,3 The binding of these antibodies to AChR activates the complement cascade, another part of the immune system, which leads to localized destruction of the neuromuscular junction.^9-11

Symptoms of gMG^5-8

Drooping of one or both eyelids
Blurred or double vision
Slurred speech
Difficulty swallowing or choking
Shortness of breath
Weakness in the arms, hands, fingers, legs, and neck

References:

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 September 25, 2019.
Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009;8(5):475-490.
Sathasivam S. Diagnosis and management of myasthenia gravis. Wiley Online Library website. https://onlinelibrary.wiley.com/doi/pdf/10.1002/pnp.315. Accessed September 25, 2019.
Silvestri N, Wolfe G. Treatment-refractory myasthenia gravis. J Clin Neuromuscul Dis. 2014;15(4):167-178.
Howard J. Targeting the complement system in refractory myasthenia gravis. Supplement to Neurology Reviews. February 2016.
Sanders DB, Wolfe GI, Benatar M, et al. International consensus guidance for management of myasthenia gravis: executive summary. Neurology. 2016;87(4):419-425.
Conti-Fine, Milani M, Kaminski HJ. Myasthenia gravis: past, present, and future. J Clin Invest. 2006;116(11):2843-2854.
Tüzün E, Huda R, Christadoss P. Complement and cytokine based therapeutic strategies in myasthenia gravis. J Autoimmun. 2011;37(2):136-143.
Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia gravis: beyond diagnosis? Expert Rev Clin Immunol. 2012;8(5):427-428.

It was a relief to have an answer. Now I knew why I was feeling what I was feeling, so we could make a plan.”

LIZ LIVING WITH gMG

HEAR HER STORY