City medical team saves Vietnamese baby with rare disease
The Children's Hospital of Fudan University has announced the successful treatment of a Vietnamese child with spinal muscular atrophy type 1, a serious and rare disease.
The baby girl was discharged on Saturday after a three-month treatment, indicating a new improvement in rare disease treatment as well as international medical tourism, the hospital said.
The baby's parents contacted the hospital for help and it provided immediate support and arrangement to travel to Shanghai.
According to the parents, the baby started to show symptoms such as weakness of the limbs and was diagnosed with spinal muscular atrophy type 1, or SMA type 1. It is an extremely severe rare disease with quick development, including progressive muscle weakness, respiration difficulty, swallowing difficulty. Most children can die of respiration failure before the age of two.
SMA is a rare progressive disease characterized by the degeneration and loss of lower motor neurons, which leads to muscle atrophy. The disease is divided into four subtypes and type 1 is the most severe form and a leading genetic cause of infant death.
Facing the baby's quick deterioration, the parents contacted with the hospital in despair, and the hospital gave a quick response and full support to streamline the process.
When the baby arrived at the hospital, she was three months old. The multidisciplinary team on SMA conducted group consultation immediately and formed an individualized treatment plan. However, the patient's condition was far more complicated than expectation.
She had suffered serious complications such as severe pneumonia and respiration failure and was sent to intensive care unit.
After emergency treatment, she was removed from the respiration machine and resumed SMA treatment.
Doctors from different departments renovated and perfected the treatment plan continuously to enhance treatment effects and quality. They had the full trust and cooperation of the parents after giving detailed explanations. The good doctor-patient relationship provides support for the baby's improvement, hospital officials said.
Doctors said the child showed positive improvement and has now regained self-respiration, while both upper limbs have regained mobility and can move freely.
After being discharged, the patient will stay in Shanghai for a period for time for regular checks and rehabilitation.
The hospital said it has participated in much high-quality and multi-center clinical research on SMA's innovative gene therapy and new drug study. It has also led a gene replacement therapy on SMA, raising China's influence on SMA treatment in the world and bringing more hope for patients and their families.
In addition, the hospital has also pushed the standardization and cooperation of SMA treatment in China. It has led the way to establish a SMA diagnosis and treatment network with the participation of 81 hospitals in 29 provinces, municipalities and autonomous regions and issued a clinical guidance to regulate and instruct SMA clinical practice in the nation.
The successful treatment of the Vietnamese baby is one more example of the hospital's capability and also a success of international medical care, which is enhanced by local government and health industry, officials said.
If you require international consultation and medical tourism with the hospital
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