Good news has emerged from the Children's Hospital of Fudan University in the field of international medical tourism. The multidisciplinary team (MDT) for spinal muscular atrophy (SMA), led by Professor Wang Yi, has successfully treated a Vietnamese infant with Type 1 SMA. After three months of meticulous treatment and dedicated efforts, the infant was successfully discharged on September 28, 2024, marking a new chapter in the hospital's rare disease treatment and international medical collaboration.
The parents of this Vietnamese infant with Type 1 SMA contacted the SMA Diagnosis and Treatment MDT team at the Children's Hospital of Fudan University after extensive efforts. The team promptly provided positive feedback. With multi-departmental support, logistical challenges for international patients seeking care in China were efficiently resolved, allowing the family to embark on their journey to China with renewed hope.
The Vietnamese infant presented with generalized hypotonia (flaccid limbs) just over one month after birth. Genetic testing confirmed Type 1 SMA, an extremely severe and rapidly progressive rare disease characterized by progressive muscle weakness, respiratory distress, and dysphagia. Most affected children succumb to respiratory failure before the age of two. Facing rapid clinical deterioration, the desperate family reached out to the Children's Hospital of Fudan University and received an immediate, comprehensive response from the team.
Upon hospital admission at 3 months old, the SMA MDT conducted a consultation and developed a personalized integrated treatment plan. However, the infant's condition proved more complex than anticipated, with severe complications including severe pneumonia and respiratory failure necessitating emergency transfer to the intensive care unit (ICU). Under the vigilant monitoring of the ICU team and close multidisciplinary collaboration, the infant overcame multiple critical episodes, was successfully weaned off mechanical ventilation, and transitioned to a clinical research ward for continued management. Adhering to principles of precision medicine, the medical and nursing teams provided holistic care. They organized multidisciplinary consultations (involving neurology, rehabilitation, pulmonology, infectious diseases, nephrology, gastroenterology, and radiology) to address persistent clinical challenges, integrate expert opinions, and continuously optimize the management plan to facilitate recovery.
Throughout the treatment, the healthcare team maintained exceptional diligence and compassion, patiently addressing the family's concerns and earning their full trust and cooperation. This strong physician-patient relationship significantly contributed to the recovery process. Ultimately, through the concerted efforts of the entire hospital team, the Vietnamese SMA infant recovered from the disease, achieving a remarkable transition from ventilator dependence and tetraplegia to successful liberation from mechanical ventilation, with restored upper limb mobility and spontaneous movement. This success not only demonstrates the hospital's clinical excellence and international influence in SMA management but also sets a new benchmark for international medical tourism.
The Children's Hospital of Fudan University remains at the forefront of SMA care. It has participated in multiple global, high-quality, multicenter clinical trials of innovative gene therapies, small molecule drugs, and antisense oligonucleotide (ASO) therapies (such as Nusinersen and Risdiplam). Related research findings have been published in top-tier international journals including The New England Journal of Medicine and The Lancet Neurology. The hospital also leads research projects on SMA gene replacement therapy, enhancing China's global standing in SMA treatment and offering new hope to patients. Furthermore, the team actively promotes standardized SMA diagnosis/treatment and multidisciplinary collaboration in China. In 2018, it spearheaded the establishment of the SMA Diagnosis and Treatment Center Alliance, which now encompasses 81 member hospitals across 29 provinces, autonomous regions, and municipalities. This network has significantly advanced the dissemination and quality of SMA care nationwide, providing standardized, high-quality medical services to more patients.
To further elevate SMA management standards, the Children's Hospital of Fudan University published the Clinical Practice Guidelines for Spinal Muscular Atrophy in 2023. These guidelines offer scientific, practical recommendations to clinicians on disease-modifying therapy selection, facilitating standardized and evidence-based SMA care in China.
The successful treatment of this Vietnamese SMA infant exemplifies both the clinical strength of the Children's Hospital of Fudan University and the potential of international medical collaboration. Moving forward, the hospital will continue its patient-centered approach, strengthen international medical partnerships, and jointly advance the development of global SMA therapeutics.
