My baby Tiantian, born in November 2014, is a cheerful, sunny, confident, and smart little girl. She was diagnosed with SMA at the age of two. For many years, due to physical reasons, mobile phones were her only playmates. When she was four years old, I was surprised to find that she already knew a lot of words. It turned out that she downloaded literacy software and taught herself. It can be seen that Tiantian is a girl who is motivated and eager to go to school like normal children. Sometimes, Tiantian would ask me why she couldn't go to school? Can she go to school after she recovers from the disease? Every time I heard her say these words, my heart was like a knife. I didn't know what language to answer her. I could only hug my daughter in my arms and secretly shed sad tears. Occasionally, games are also her favorite. She has made many children with the same interests in them. Before going to bed at night, she always talks to me about what happened that day, with joy and happiness. When talking about something exciting, she always hugs me and laughs non-stop!
When my daughter was one and a half years old, my friends reminded me that Tiantian's walking posture was not right and I should go to the hospital for a check-up. At that time, I was immersed in the pain of losing my beloved son and neglected my daughter's situation. When I calmed down and observed carefully, I found that my daughter walked like a duck and could not step up a slope that was slightly higher than two centimeters from the ground. Her center of gravity was unstable and she was prone to falling. On September 30, 2016, she was diagnosed with SMA at Guangzhou Children's Hospital. At that time, the doctor told me that this was a rare disease and there was no medicine for the time being. I was told to go back and take good care of the child. She might have to sit in a wheelchair when she was seven or eight years old. I should not listen to rumors, seek medical treatment blindly, and waste money. Only when new drugs for the treatment of SMA are available can Tiantian live a normal life! The doctor's words were like a bolt from the blue! The sky collapsed again! I looked at the doctor quietly, crying silently, my mind was blank, God would not give me a way out! He took my son's life just a year ago! My spirit was completely broken! Bedridden, crying all day, the child's father was speechless and felt helpless...
First look at GeneCradle, empowering families of patients with rare diseases
In November 2016, Tiantian's father received a call from a classmate in Beijing, informing him that a new drug research and development company in Yizhuang, Beijing was developing a special drug for the treatment of SMA, and provided contact information. Finally, there is good news! We were overjoyed and immediately called Dr. Wu Xiaobing, the founder of GeneCradle. Dr. Wu answered the phone patiently and enthusiastically, and explained the current situation of the gene therapy SMA special drug. At the same time, she told us that SMA is a common disease among rare diseases. She also felt sorry for the children because she knew that there were many children with SMA! As a gene company with the ability to develop SMA treatment drugs, she felt a great responsibility. Dr. Wu resolutely faced the challenge and put all the company's efforts into the SMA project. Then, Dr. Wu comforted us and said: Don't be sad, don't worry, have confidence, believe in GeneCradle, it will definitely succeed! Suddenly, our hearts were relieved a lot, and we felt that the world was bright!The road of waiting is long and torturous
Six years later, in 2022, our family finally received good news. The SMA gene drug GC101 developed by Beijing GeneCradle was ready to start clinical trials. In June of the same year, under the careful care of GeneCradle and the staff of the Seventh Medical Center of the PLA General Hospital, Tiantian successfully underwent the first phase of the clinical trial of GC101. Less than 24 hours after taking the medicine, Tiantian said that she felt her legs and even her whole body were full of strength, and asked to help her stand up. I tried to help her stand up, and she actually stood alone for 8 seconds! The family hugged each other, laughing and crying... In the next few days, I noticed that Tiantian could lower her head and raise it again, and she no longer had to support her chin. She could lower her head and raise her head freely, and she could stand alone for a while. It was really immediate. Since then, Tiantian has made continuous progress and her physical fitness has also greatly improved. When I held her before, I always felt loose and soft. Now it is getting stronger and stronger, and it feels the same as a normal child. Before receiving GC101, Tiantian had dull eyes and a sickly look. After taking the medicine, her eyes became bright, her complexion became rosy, and she became lively and active.Belief is power, persistence is victory
It has been almost two years since she started using GC101. During this period, Tiantian had three episodes of severe pneumonia and was admitted to the intensive care unit once. She was infected with COVID-19, influenza A, and influenza B respectively. She was not affected much and did not deteriorate at all. Due to various reasons, Tiantian can only be hospitalized for a maximum of 5 months a year. The lower end of her thigh was atrophied before taking the medicine, but now she has grown muscles and is strong. She can stand alone for more than a minute. I believe that Tiantian will be able to walk soon! Our family thanks GeneCradle and wishes all SMA patients and their families: May your dreams come true! Belief is power! Persistence is victory! "