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 Genzyme 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.
In 1997, when he was 5 years old, Amr’s health inexplicably deteriorated. His family, led by his father Maamon, searched for answers anywhere they could find them.
At the time, Amr’s family was living in Amman, Jordan. His parents took him to a Jordanian blood specialist who confirmed that his mysterious illness was Gaucher disease. The doctor recommended the family seek advice from a specialist who had experience with Gaucher.
After examining Amr, the specialist told his family the support Amr would need was not available in Jordan and recommended the family seek help in Cairo, Egypt, where a new program was being developed to support patients with Gaucher.
Shortly after, Amr became the first patient admitted into Sanofi Genzyme’s Humanitarian Program in Egypt. This marked the beginning of the Gaucher Initiative, a collaboration with Project Hope, a global health and humanitarian relief organization, to provide Gaucher patients with support.
Maamon relocated his family to Cairo so Amr could receive the support he still needs. He kept his job as an accountant in Jordan and is only occasionally able to travel to Egypt to visit his family due to his work and the expense of travel.
His father’s sacrifice has helped Amr grow into a creative young man who recently got married. “I love anything creative…so I love concerts, I love dancing,” Amr says. “I took dance classes for a little period of time. I love drawing and art. I like to go to the opera house. I like to go to galleries.”
L to R: Amr, Amr’s wife, Dr. Reda Mansour, Amr’s mother, Dr. Khaled Eid
The Gaucher Initiative has supported hundreds of patients with the disease in Egypt and has helped build the capacity of the Egyptian health system to properly diagnose, refer and manage Gaucher patients. The program has also contributed to countrywide understanding of the disease.
“The Gaucher Initiative project started in Egypt at a time when Gaucher was not known even as a name. We started with 12 patients identified and no ecosystem to be able to support them,” explained Medhat El-Bialy, Head of Rare Diseases, Emerging Markets, Sanofi Genzyme. “The Gaucher Initiative came to build that ecosystem around them to be able to create a sustainable environment where these patients can receive the best of care.”
This year marked the 20th Anniversary of the Gaucher Initiative. Watch the video below to learn more about the program.