Rare diseases do not discriminate; they affect people from all walks of life and in all economic circumstances. Sanofi’s responsibility to the patient community extends beyond the research and development of effective therapies to include providing support to patients, who often have nowhere else to go–no matter who they are or where they live.
Sanofi has two Humanitarian Programs, which provide support to patients with five different lysosomal storage disorders (LSDs), a group of rare genetic conditions caused by enzyme deficiencies, as well as patients with hemophilia, a rare genetic disorder in which a person’s blood does not clot normally.
Throughout the month, we’ll be sharing stories of patients around the world who receive this support and who inspire Sanofi’s employees around the world.
When Zhengtao was diagnosed with Gaucher disease as a child in the mid-1990s, his family had never heard of the disease. At that time, no child had been diagnosed with Gaucher in China and information about the disease was extremely limited. His mother didn’t know where to turn for help.
Zhengtao, Gaucher disease, China
Gaucher disease is a rare, inherited disorder in which a lipid, a fat-like substance in the blood, accumulates in different parts of the body, primarily the spleen, liver and bone marrow. People with the disease lack sufficient levels of a particular enzyme called glucocerebrosidase. Symptoms may begin early in life or adulthood. Typically, people bruise easily due to low blood platelets and experience fatigue due to anemia. They also have an enlarged liver and spleen and skeletal disorders. Many patients may have a mild form of the disorder with very few symptoms.
After years of searching for information that could help her then three-year-old child, Zhengtao’s mother stumbled upon Sanofi’s website. She sent an email to the company with a message along the lines of: “My son has Gaucher disease. We live in China. Can you help us?”
That message eventually led to Zhengtao becoming the first Gaucher patient to be diagnosed in China when he was nine years old. Now 31, Zhengtao is the father of two children and happily lives with his wife, children and mother. He still receives humanitarian support through Sanofi’s China Charitable Access Program.
Zhengtao was the first patient supported by the China Charitable Access Program, which began in 1999 and has grown to provide humanitarian support to hundreds of Gaucher patients in China. Sanofi currently provides support to more than 100 Gaucher patients in the country and recently celebrated the program’s 20-year anniversary.
Since the China Charitable Access Program was established, the Chinese healthcare system has evolved. In 2016, the Chinese government launched Healthy China 2030 to bring access to healthcare to each of its 1.4 billion inhabitants.
As part of this plan, China released the China Rare Diseases Research Report (2018), which included the first official list of 121 recognized rare diseases with the aim of improving access to information and sustainable support for its rare disease patients. China has also seen an increase in the formation of patient advocacy organizations, which provide educational and peer support to patients.
“By promoting Sanofi’s China Charitable Access program, we have aroused the attention of people from all walks of life about rare diseases,” said Brian Zhiwei, Secretary General of China Charity Federation.
Zhengtao founded the first Gaucher disease patient advocacy organization in China, which now has more than 200 members, and also started the Firefly Choir, composed entirely of rare disease patients.
“In 2013, we conducted our first concert in Beijing, and over 500 people came to watch our performance,” Zhengtao said. “During the curtain call, we cried – tears not for the disease and pain we have gone through, but for the hope that we always bear in mind.”
SAGLB.GZ.19.10.1561a - Approved October 2019