J&J at AANEM 2024: Recognizing pediatric unmet need in generalized myasthenia gravis

This week, I am in Savannah, Georgia for the 2024 Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, where our J&J team has a clear objective: To present new research on generalized myasthenia gravis (gMG) in adolescents as well as adults that we hope may one day help to improve the lives of people living with this debilitating condition.

Myasthenia gravis (MG) is a chronic, life-long, and rare autoantibody-driven disorder in which antibodies destroy the communication between nerves and muscle, resulting in weakness of the skeletal muscles. MG can progress into a more serious form, called generalized myasthenia gravis or gMG, which impacts more than 100,000 people in the U.S.[1],[2] Imagine: one day, you slowly start to lose control over your eyes, mouth, throat and limbs. Your vision blurs. Your speech slurs. You begin having difficulty chewing, swallowing and breathing.[3],[4]

Adolescents (12 – 17 years old) represent about 10 percent of all new MG diagnoses and have clinical features that are often more severe than the adult experience.[5],[6],[7],[8]?Starting even before diagnosis, gMG can be tricky to pinpoint in pediatric populations. As often happens in children and adolescents, gMG symptoms such as fatigue and muscle weakness may be written off as benign causes such as stress or growth spurts. It’s not uncommon for patients to visit multiple specialists before finally receiving a diagnosis.[3],[4]?Understandably, this delay can lead to feelings of confusion and helplessness or that their symptoms aren’t being taken seriously.

As you can imagine, this disorder can have a serious impact on a teenager’s social life, mental health as well as many other aspects of their life and wellbeing. It may affect mobility, which can impact their ability to participate in sports or even just spend time with friends. Some adolescents even experience increased bullying from peers. The fluctuating nature of their symptoms can lead to missed school days, which not only affects academics but also self-esteem.[3],[4] Adolescents with severe forms of gMG may even need a ventilator to breathe or a feeding tube due to the extent of the symptoms, which can have significant quality of life implications.[9]

Currently, other than symptom management options, there are no approved advanced therapies in the U.S. that may address the root cause of gMG in adolescents.[10] In the clinical setting, my colleagues and I have encountered dozens of patients with gMG who are eager for new treatments that allow them to help manage symptoms and live fulfilling, active lives.

At AANEM, J&J is excited to be presenting data that we hope brings us one step closer by sharing new research which may help address unmet need for people with gMG. I am proud to be working to help address these challenges not only for the entire gMG patient population, but especially teens, who deserve therapeutic options to help manage their gMG.

To learn more about the work for autoantibody-driven disorders, and our presence at AANEM this year: please visit our Newsroom.

[1] Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health - Western Pacific. https://www.thelancet.com/action/showPdf?pii=S2666-6065%2820%2930063-8

[2] DRG EPI (2021) & Optum Claims Analysis Jan 2012-December 2020.

[3] Myasthenia Gravis Association. Teen’s experience with Myasthenia Gravis. Available at: https://www.youtube.com/watch?v=77ituJKch_g . Last accessed: September 2024.

[4] WebMD. My Daily Life with Myasthenia Gravis. Available at: https://www.webmd.com/brain/features/myasthenia-gravis-life-with . Last accessed: September 2024.

[5] Evoli A, Batocchi AP, Bartoccioni E, Lino MM, Minisci C, Tonali P. Juvenile myasthenia gravis with prepubertal onset. Neuromuscul Disord. 1998 Dec;8(8):561-7. doi: 10.1016/s0960-8966(98)00077-7.

[6] Evoli A. Acquired myasthenia gravis in childhood. Curr Opin Neurol. 2010 Oct;23(5):536-40. doi: 10.1097/WCO.0b013e32833c32af.

[7] Finnis MF, Jayawant S. Juvenile myasthenia gravis: a paediatric perspective. Autoimmune Dis. 2011;2011:404101. doi: 10.4061/2011/404101.

[8] O'Connell K, Ramdas S, Palace J. Management of Juvenile Myasthenia Gravis. Front Neurol. 2020 Jul 24;11:743. doi: 10.3389/fneur.2020.00743.?

[9] Stanford Medicine Children’s Health. Myasthenia Gravis (MG) in Children. Available at: https://www.stanfordchildrens.org/en/topic/default?id=myasthenia-gravis-mg-in-children-90-P02612 . Last accessed: September 2024.

[10] Finnis MF, Jayawant S. Juvenile myasthenia gravis: a paediatric perspective. Autoimmune Dis. 2011;2011:404101. doi: 10.4061/2011/404101.