This is part of a series recognizing the 25th anniversary of Sanofi Genzyme’s Humanitarian program. The primary goal of our Humanitarian program is to deliver enzyme replacement therapy, to the best of our ability, to patients with lysosomal storage disorders (LSDs) who have a demonstrated need in certain circumstances where treatment access is limited. This series highlights our humanitarian efforts to support the LSD community, and represents just one part of Sanofi and Sanofi Genzyme’s charitable work.
Since the beginning of our organization, we recognized the need to help patients access our therapies. We understood that creating an effective treatment doesn’t mean anything if it can't reach those who need it, and we made a commitment to help lysosomal storage disorder patients with a demonstrated need in certain circumstances where treatment access is limited. The first patients received access to free therapy within our first year of having a commercial product -- in 1991 in the United States. These are three patient stories that illustrate the beginnings of our humanitarian programs in Egypt, China and India.
Amr, Gaucher disease, Egypt
Maamon and his family were living in Aman, Jordan in 1997 when his son, Amr’s inexplicable deteriorating health led them to look for answers anywhere they could. A Jordanian blood specialist confirmed that the mysterious illness afflicting their son was Gaucher disease and recommended that they seek the counsel of a doctor in Jerusalem who had experience with it. Dr. Zimran, an early treater of Gaucher disease, examined Amr and told the family that he needed an enzyme replacement therapy that was unavailable to them in Israel. The doctor did, however, hear about treatments being provided in Cairo, Egypt through a new program and helped Amr’s family make contact with Genzyme to petition for help. Shortly after, Amr became the first patient admitted into Sanofi Genzyme’s Humanitarian program in Egypt.
This was the beginning of the Gaucher Initiative, a collaboration with Project Hope to provide Gaucher patients with access to therapy regardless of their ability to pay. Since its inception, hundreds of patients in Egypt have received treatment free of cost. Additionally, the program has helped build the capacity of the Egyptian health system to properly diagnose, refer and manage Gaucher patients, and has contributed to countrywide understanding of the disease.
Zhengtao, Gaucher disease, China
In 1998, after years of searching for information and hope for her family and her three-year-old son who had been diagnosed with a disease she'd never heard of before, a mother stumbled upon Genzyme’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 send us medicine?”
Zhengtao became the first Gaucher patient treated in China when he was nine years old. Now 28 years old, Zhengtao is still a humanitarian patient in Sanofi Genzyme’s China Charitable Access Program, which currently provides treatment to more than 100 Gaucher patients. Zhengtao is also the founder of China’s first Gaucher disease patient organization and started the Firefly Choir, composed entirely of rare disease patients.
Suyog, Gaucher disease, India
Suyog’s family felt hopeless when they learned he had Gaucher disease, a rare disease that was virtually unknown in India in 1999. By this point, his liver and spleen had swollen considerably, and he had difficulty breathing. When they learned treatments existed, his parents wrote to Genzyme to see if there was any way assistance could be provided to their son. They were delighted to hear back with the news that their son would be receiving treatment free of charge. Suyog completed his engineering degree last year and currently works at an IT firm.
Sanofi Genzyme’s India Charitable Access Program (INCAP) has provided treatment at no cost to Suyog and other patients who are not covered by insurance and lack the financial resources to obtain treatment. Formally established in 2007, INCAP now treats more than 100 patients, and has made strides in improving awareness, diagnostics, and management of patients suffering from LSDs in India.
Today, the humanitarian programs in Egypt, China and India make up almost half of our humanitarian efforts around the world. We’ve established country specific programs in these markets managed by a team of local medical experts that work to ensure the programs meet the unique needs of the patients we serve.
Over the last two decades, our humanitarian programs have evolved and expanded to now support five different lysosomal storage disorder communities around the world. “These are only the stories of three patients,” said Simone Azevedo, Sanofi Genzyme’s Global Head of Rare Hematology. “However, they exemplify the 1,700+ patients in 70 countries around the world that have received free therapy through our humanitarian programs over the past 25 years.”