RARE DISEASES September 23, 2021 The Sanofi Genzyme Rare Humanitarian Program Turns 30 Facebook Twitter LinkedIn Mai Hoa from Vietnam has a rare disease called Mucopolysaccharidosis I, a genetic condition caused by enzyme deficiencies For three decades, Sanofi has provided humanitarian support to people living with rare diseases worldwide. This is a vital part of our mission to improve lives. Founded in 1991, Sanofi Genzyme’s Rare Humanitarian Program was created by Henri Termeer to help put patients at the center of care. A visionary in supporting people with rare diseases across the globe, Termeer believed that creating medicines was not enough and more had to be done to help those who could not afford or access treatment. “Our purpose is clear–to help ensure that people living with rare diseases around the world have the ability to access the treatments they need,” said Paul Hudson, Chief Executive Officer. “Henri saw this unmet need 30 years ago. And now, we are honored to continue his important work through our Rare Humanitarian Program. But our responsibility does not end with developing effective therapies. We are ensuring sustainable access to approved treatments for patients with rare diseases, regardless of their ability to pay when they meet the program’s criteria.” The program was the first humanitarian initiative for people with lysosomal storage disorders (LSDs)–a group of rare genetic conditions caused by enzyme deficiencies, including Gaucher disease, Fabry disease, Pompe disease, and mucopolysaccharidoses (MPS) I and II–and is the longest running program of its kind. The Impact of the Humanitarian Program over 30 Years Putting patients first Over the past 30 years, our program has helped improve the health and lives of thousands of people with rare diseases across the globe. In addition to providing access to free therapy for patients who meet the program’s criteria, we have offered support for patient diagnosis, treatment monitoring, patient advocacy and physician education. The Rare Humanitarian Program is a key component of these activities and works hand-in-hand with teams across Sanofi Genzyme to create sustainable treatment for people with LSDs. An important part of supporting rare disease communities is understanding. Through the program, Termeer met with patients and families to get to know them, their stories, and the challenges they faced. And today, Sanofi Genzyme teams around the world carry on Termeer’s legacy by listening to the needs of our patients and gaining on-the-ground insights, particularly around the obstacles facing a country and its rare disease community. It’s these personal stories that inspire our work every day and push us to do more. Hear from some of the people whose lives have been changed through the program: Amr, living with Gaucher disease, from Egypt, with Dr. Khaled Eid, Dr. Reda Mansour and Amr’s mother, Somaya Amr and Dr. Mansour’s Story "When the program started, no one with Gaucher disease had ever been treated in Egypt. Now, there are eight centers across the country and clinical specialists who are experts in the treatment and management of this debilitating disease. Now, we are able to offer support to the very ill." Dr. Reda Mansour, Chief of Party, Project HOPE Dr. Reda Mansour was one of our earliest partners and has been a champion for Gaucher disease patients for over 30 years. In 1998, Amr became the first patient with Gaucher disease to receive treatment in Egypt, paving the way for many current and future patients affected by this rare LSD. Allyson, living with Pompe disease, from New Zealand, with her husband, Steve Allyson’s Story "I got to the point where I could only have a shower or make dinner for the family. I could only do one of those things per day. I was bed-ridden. That’s all the energy I had. I asked Sanofi Genzyme to help and said, ‘I know you don’t owe us anything, but please help if you can.’ You have no idea how grateful I am." Allyson, Patient and Patient Advocate Allyson is one of 11 people living with Pompe disease in New Zealand. Today, she spreads awareness as the head of the New Zealand Pompe Patient Organization by actively sharing her story with the goal of helping others in a similar situation. Camila, living with MPS I, from Ecuador Camila and Dr. Velez’s Story "When I’m asked why I got involved with rare diseases, my answer is always the same: it is because of Camila. For more than 10 years the two of us have walked together, see each other grow. I have seen her become a 14-year-old girl who is a warrior. She has faced the diagnostic odyssey of all patients with rare disease and she spreads optimism with her tireless desire to stay healthy." Dr. Paola Vélez Pinos Dr. Velez has been supporting patients with rare diseases in Ecuador for many years, sharing stories of courage, dedication, and strength from the people she helps. She is a dedicated front-line fighter, and her commitment to people with rare diseases shines through her tireless conviction to helping people like Camila live normal lives. For the past 30 years, we have been honored to help patients living with LSDs gain access to the treatment they need and to supporting local communities build sustainable healthcare systems. Being a trusted partner to the global rare disease community is at the heart of our work. Bill Sibold, Executive Vice President and Global Head, Sanofi Genzyme Please click here to read more inspiring stories from the Rare Humanitarian Program. MAT-GLB-2103311 v0.2 08/2021 Follow us Sanofi on Twitter Sanofi TV on YouTube Sanofi On LinkedIn Sanofi on Instagram Sanofi RSS feeds Contact Legal notice Privacy & Data Protection Cookies policy Corporate code of ethics Our publications Sitemap Our websites Accessibility: partial compliance © Sanofi 2004-2021 - All rights reserved