Breaking the “Fluctuating” Dilemma of Generalized Myasthenia Gravis: Multi-Stakeholder Call for Innovative Treatments to Benefit More Patients

BeijingJune 15, 2026 /PRNewswire/ — Recently, a science popularization roundtable on the theme of Generalized Myasthenia Gravis (gMG) was held in Beijing, accompanied by the premiere of the patient-themed short film “Breaking Waves, New Life.” At the event, multiple clinical experts, pharmacoeconomics scholars, patient organization representatives, and corporate representatives gathered to engage in in-depth discussions on core issues such as disease burden, standardized diagnosis and treatment, and medication security for children. They jointly called on all sectors of society to increase attention to the long-term needs of gMG patients, helping patients stabilize fluctuations and reshape their lives.

Professor Zhao Zhongbo, Vice President of Huashan Hospital Affiliated to Fudan University and Chief Physician of the Department of Neurology, stated: “The most characteristic manifestation of gMG lies in the repeated fluctuations of whole-body strength, ‘lighter in the morning, heavier in the evening,’ pushing patients into a passive life of loss of control, severely depriving them of their social functions, and plunging the entire family into the heavy burden of long-term care and financial strain. For children and adolescents in the critical stage of growth and development, this fluctuation not only drains their current life but also their future—irreversible interference with physical development and sustained impact on mental health. We hope to take this opportunity to unite forces from clinical, social, and policy sectors, enabling more patients and families to escape this predicament of fluctuations.”

The Truth of “Lighter in the Morning, Heavier in the Evening”: Seeing the Childhood and Life Drained Behind the Fluctuations

MG is a complex chronic autoimmune disease characterized by acquired transmission disorders at the neuromuscular junction (NMJ)^[1]. gMG, as a type primarily involving systemic muscle groups, is most characteristically manifested by fluctuating weakness and easy fatigability of skeletal muscles. Patients’ daily symptoms often show dynamic fluctuations of “lighter in the morning, heavier in the evening,” worsening after activity and improving with rest^[2]. This elusive sense of weakness makes the patient’s physical state constantly uncertain, frequently leading to a loss of control over life.

gMG can occur at any age. For children and adolescent patients, the physical and mental burden brought by the disease is particularly severe: as the disease progresses, patients may develop dysphagia and dysarthria. Among those who develop refractory cases, the risk of acute exacerbation and myasthenic crisis increases by 4.7 times and 4 times, respectively, and can lead to death in severe cases^[3].

Beyond the heavy physical burden, the psychological trauma caused by the disease cannot be ignored. In the patient-themed short film, patient Fanfan (pseudonym) shared the psychological burden she carried due to the disease in her childhood: “My memories of childhood almost all begin with the disease. When I was young, as long as I heard there was a hospital or a doctor who might cure this disease, my parents would immediately take me there. Seeking medical treatment, referrals, and traveling had become part of the whole family’s life. As I grew older, I was even chased by all the boys in my class and given nicknames. I was very afraid of others asking me why I was like a monster.”

Professor Yin Jian, Chief Physician of the Department of Neurology at Beijing Hospital and the National Center for Geriatric Medicine, pointed out: “In daily clinical diagnosis and treatment, we truly see the immense physical and mental burden that gMG imposes on pediatric patients and their families. Children are in a critical stage of growth and development. The repeated fluctuations of the disease not only severely limit their daily learning and social activities but also have a sustained and profound negative impact on their physical and mental development. Faced with such a heavy disease burden, breaking the vicious cycle of disease recurrence is a key clinical challenge that urgently needs to be addressed. Fortunately, recent novel targeted biologics have benefited pediatric patients, bringing new hope to fill the unmet clinical needs of children with gMG. We look forward to these innovative therapies further improving clinical accessibility, helping more pediatric patients truly escape the predicament of fluctuations and regain opportunities for healthy growth.”

Breaking the Predicament of Fluctuations: The “Dual Goal” Strategy Leading a New Direction in Treatment

For a long time, the clinical treatment of gMG has primarily relied on traditional methods such as cholinesterase inhibitors, glucocorticoids, non-steroidal immunosuppressants, intravenous immunoglobulin, plasma exchange, and thymectomy to improve the condition^[4]. However, due to the prolonged course of gMG, a large number of patients still frequently experience disease fluctuations or exacerbations after receiving traditional treatment^[5],[6]. Particularly noteworthy is that to suppress fluctuating symptoms, some patients have to use traditional drugs like hormones long-term or in high doses.

With the development and widespread application of novel targeted biologics, the diagnosis and treatment concept for gMG has evolved from simple symptom control to a “dual goal” strategy focusing on long-term disease management. Professor Zhao Zhongbo stated: “As the diagnosis and treatment concept continues to advance, the clinical field is increasingly focusing on how to effectively control symptoms and reduce disease fluctuations while also considering patients’ long-term quality of life and treatment safety. Especially for pediatric patients, treatment should not only focus on improving current symptoms but also comprehensively consider growth and development, mental health, and future life. Modern gMG management is moving from simple symptom control towards more stable, precise, and sustainable long-term management, helping patients achieve fewer fluctuations and relapses and return to normal life as much as possible.”

Helping Children with gMG Strive for a Stable Future: Multiple Parties Call for Improving Medication Security for Children

The impact of gMG on children extends far beyond the symptoms themselves—repeated fluctuations disrupt academics, psychology, and social integration, consuming growth time and placing long-term care pressure and financial burden on families. The limited treatment options further exacerbate this situation.

Approximately 10% to 20% of pediatric patients progress to refractory gMG^[7], with the risk of acute exacerbation increasing by 4.7 times and the risk of crisis by 4 times compared to non-refractory cases, potentially leading to death in severe cases^[8]. In terms of treatment, long-term hormone therapy has profound effects on pediatric patients: the 3-year remission rate with conventional immunotherapy is only 48.6%^[9], the incidence of glucocorticoid-induced MG exacerbation is about 33.3%, and if it progresses to severe respiratory or swallowing difficulties, emergency ICU care may be required^[10]; 87% of pediatric patients have a height below their target height^[11], and it can cause severe adverse reactions such as obesity, secondary diabetes, bone marrow suppression, and liver and kidney function damage^[12]-[18]. The combination of disease fluctuations and treatment side effects not only long-term affects the academic, psychological, and social development of pediatric patients but also imposes a long-term heavy economic and care burden on families.

Professor Zhang Fang, Chairman of the Drug Economic Evaluation Branch of the China Medical Education Association, stated: “The burden borne by families of children with gMG goes far beyond direct medical expenses. Hospitalization, long-term care, parental work absenteeism, and costs of secondary diseases together constitute a severely underestimated social cost. In recent years, the country has actively explored pathways for ensuring medication access for children and rare diseases, with relevant policy signals continuously being released. We look forward to the advancement of this process, promoting the accessibility and payment security level of innovative drugs for pediatric gMG, so that every child patient can obtain treatment opportunities that can change their destiny. Children are the future that society cannot afford to disappoint. The whole society is joining hands to build a solid protective wall for gMG patients and their families—disease can limit the body, but it can never stop the future paved by love and hope for children.”

The accessibility of innovative drugs is not just a policy issue; it concerns whether the future of these children can truly be supported. “When people and families are long-term trapped by the disease, life seems to have no end, like a dark tunnel without an exit.” Qing Zhao, Founder and Director of the Beijing Ailijian Rare Disease Care Center for Myasthenia Gravis, emotionally said: “Over the years, Ailijian has accompanied many children with generalized myasthenia gravis through long journeys of seeking medical treatment—some children started running to hospitals as soon as they learned to walk, and some families traveled thousands of kilometers for a single consultation. What we see is the disease constantly taking away the growth time they should have had. Children with generalized myasthenia gravis are at the starting point of their lives, but they face a fluctuating tug-of-war that may affect their entire life cycle. We urgently call for myasthenia gravis to be included in the scope of special or major disease coverage in various regions, and for novel targeted biologics for pediatric generalized myasthenia gravis to be included in medical insurance as soon as possible. This is the common expectation of these children’s families.”

Hu Yiqing, Vice President of AstraZeneca China and Head of the Rare Disease Business Unit, stated: “gMG patients, especially pediatric patients, still face real challenges such as repeated disease fluctuations, prominent unmet treatment needs, and insufficient accessibility. AstraZeneca has always been patient-centered, continuously promoting R&D innovation and accessibility for rare disease treatments, striving to transform cutting-edge scientific achievements into treatment options that are usable and affordable for patients. We also look forward to working with clinical experts, academic institutions, patient organizations, and relevant parties to jointly promote medication security for children with gMG. With the power of science, we illuminate rare new lives and reshape the pillars of society.”