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Rare yet devastating
The quality of life of a patient living with a rare disease may be severely limited due to loss of autonomy and the progressive, debilitating aspects of the disease. The rarity and complexity of patients’ medical conditions can make a rare disease difficult to diagnose and treat, leaving patients feeling isolated. Fortunately, thanks to innovations in biotechnology there are an increasing number of targeted therapeutic treatments available. Beyond this, reaching out to fellow patients is crucial in providing individuals and their families with psychological support and hope.
Within Sanofi Genzyme’s Rare Diseases Global Business Unit, our core activities are currently focused on two medical areas: Genetic Diseases and Endocrinology. Lysosomal Storage Disorders (LSDs) - a group of rare genetic conditions - are a cornerstone of Sanofi Genzyme's business, and the medical area for which we are most well-known. LSDs are a group of more than 40 diseases. Each is caused by a genetic problem that results in the deficiency or malfunction of particular enzymes needed to remove waste material from cells. These waste molecules then accumulate, or are stored, in cell lysosomes (smaller compartments within the cells), disrupting cell function and causing a variety of symptoms.
Living with a Rare Disease
Fuensanta and Ramon Eulacio talk about their three children with Gaucher disease and the comfort they have found working closely with Sanofi Genzyme.
As a pioneer and leader in enzyme replacement therapies, Sanofi Genzyme is committed to finding new therapeutic solutions for patients who may have no other alternatives. We pioneered the first enzyme replacement therapy to treat Gaucher disease in 1984, transforming the lives of patients and at the same time setting the standard for treating the condition.
Our breakthrough research in genetic engineering and recombinant protein manufacturing has enabled us to develop targeted therapies for several previously untreatable LSDs, including Gaucher disease, Fabry disease and Pompe disease. More recently we began clinical trials for an investigational therapy for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B. This process took the form of translational medicine, with an in-depth investigation into the underlying causes of the disease and the needs of the patient community.
Though it has taken years of commitment from scientists to begin realizing the potential of gene therapy, we believe it will lead to transformational applications for patients in need of treatment options.
Strength in collaboration
Sanofi Genzyme’s scientists collaborate with external partners to develop groundbreaking new therapies for patients who might otherwise have few or no treatment options.
Our alliance with biopharmaceutical company Alnylam is a case in point. Together, we are developing novel therapeutics based on gene splicing, or RiboNucleic Acid (RNA) interference, which works by inhibiting the production of proteins that cause or contribute to the targeted disease.
Engaging with patient communities
“We differentiate ourselves by some of the most basic, fundamental things: taking the time to listen and really trying to understand the needs of patient communities.” David Meeker, Executive Vice President, Head of Sanofi Genzyme.
Sanofi Genzyme has been an active partner with the communities we serve for over three decades. Driven by our commitment to patients, we strive to develop strong relationships with patient communities and listen to their perspectives so that we can truly understand their needs. These collaborations guide us as we continue to push the boundaries of medicine and technology to develop new and better therapies where none existed before.
In 2001, we established our Patient Advocacy group to formalize our collaborations with the advocacy community. Since then we have supported the work of countless patient organizations around the world. We believe our diverse efforts not only help individual groups, but can also build bridges across different disease communities so that they and their leaders may learn from one another’s experiences.
For five consecutive years, we have held the Patient Advocacy Leadership (PAL) Awards grant program for non-profit organizations that serve the LSD patient community. It seeks to spark innovation in disease awareness programs and patient support initiatives around the world. We also host Global Advocacy Leadership Summits that bring together leaders from within the rare disease community to network with one another as well as interact with Sanofi Genzyme staff and executives.
Sanofi Genzyme, the Spirit of Hope
For the availability of our healthcare solutions in your country, please consult your local Sanofi website.
Engaged with patients for over 30 years, Sanofi Genzyme led the discovery of the first therapies targeting rare genetic diseases.