A 2-year-old Swedish girl has been reborn in Guangzhou after treatment for a rare disease.

A 2-year-old Swedish girl has been reborn in Guangzhou after treatment for a rare disease.

On June 20, Juan Carlos (translated) from Sweden came to Guangzhou with his 2-year-old daughter Alma to seek medical treatment because the child was suffering from the rare disease “Metachromatic Leukodystrophy (MLD )””. Guangzhou Medical University Women’s and Children’s Medical Center (hereinafter referred to as “Guangzhou Women’s and Children’s Center”) used gene therapy to treat Alma, and the child was successfully discharged from the hospital on October 25. Experts said that due to timely treatment, it is expected that the child will be able to return to a normal life in the future.

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Swedish baby girl Alma still has trouble standing and has an unsteady walk at 1 year old In May this year, Alma was first diagnosed with metachromatic leukodystrophy by a local hospital in Sweden. When he discovered that his child had a rare disease that was difficult to treat, Alma’s father, Juan Carlos, a nurse, began looking online for treatments related to MLD. But Alma’s disease cannot be treated locally in Sweden, so Alma’s father uses the internet to find clues all over the world.

In early June this year, Juan Carlos saw a research paper on the Internet by Professor Lian Qizhou from the School of Synthetic Biology at Shenzhen University of Technology, and found that the school had collaborated with the Guangzhou Women and Children’s Center to conduct medical research. who had successfully treated 6 people. The sick child received gene therapy.

On June 16, Juan Carlos had an online video chat with Director Jiang Hua of the Department of Hematology and Oncology of Guangzhou Women and Children’s Center, Lian Qizhou’s team and Jiang Hua’s team made a detailed assessment of Alma’s condition and provided an initial plan, ” We believe that the child has a chance to be treated,” said Dr. Jiang Hua.

Gene stem cells were successfully transplanted within a month

The opportunity is rare and treatment is willing. Four days later, on June 20, Juan Carlos rushed to the outpatient clinic of Director Jiang Hua of the Guangzhou Women and Children’s Center for a consultation. Jiang Hua said that based on the medical history and laboratory tests, it was determined that Alma was in an early stage and there were possibilities for treatment.

Existing treatment strategies include ARSA enzyme replacement therapy, adeno-associated virus gene therapy, and allogeneic hematopoietic stem cell transplantation. After extensive deliberation, the hospital decided to give the child adeno-associated virus gene therapy.

After collecting hematopoietic stem cells, Alma was admitted to the hospital for a stem cell transplant on September 26. After myeloablative treatment, on October 9, doctors implanted the edited autologous stem cells into Alma’s body. This process is not easy and wait for new stem cells to grow and rebuild hematopoiesis, continuously Make cells with enzymatic activity to cure diseases.

After staying in the transplant warehouse for a full 28 days, Alma was transferred to the general ward. On October 25, Alma’s neutrophils were infused, her platelets were stable, and there was no infection.

“We really hope that this treatment will be successful. If she can grow up smoothly, we will bring her back here and show her the hospital that gave her a new life,” said Alma’s father.

Alma’s father was full of praise for Guang Fu’s treatment and care afterwards “Although it was a very difficult time, we felt safe here and we were not alone. Everyone was someone help us. My daughter has now fallen in love with eating Chinese noodles.”