SAN DIEGO (February 28, 2017) – Retrophin, Inc. (NASDAQ: RTRX) today joins patients, advocates, healthcare professionals, researchers, public health authorities and the biopharmaceutical industry in recognizing Rare Disease Day. This annual global observance seeks to raise awareness of rare diseases and their impact on the lives of people living with these disorders, including an estimated 30 million people in both the U.S. and Europe.i
In the U.S., diseases affecting fewer than 200,000 patients, and fewer than 1 in 2,000 patients in the EUii, are considered rare. Approximately half of all rare diseases affect children, and 80 percent of these conditions have genetic originsiii.
“Retrophin is proud to be a part of the rare disease community that is united in its efforts to shed light on the significant burden faced by people living with rare diseases, and their need for treatment options,” said Stephen Aselage, chief executive officer of Retrophin.
Rare Disease Day is led internationally by the European Organisation for Rare Diseases (EURORDIS) and in the U.S. by the National Organization for Rare Disorders (NORD). For more information, visit http://www.rarediseaseday.org/ or www.rarediseaseday.us.
EURORDIS is a non-governmental patient-driven alliance of 606 rare disease patient organizations in 56 countries. EURORDIS strives to build a strong pan-European community of patient organizations and people living with rare diseases, to be their voice at the European level, and - directly or indirectly - to fight against the impact of rare diseases on their lives. EURORDIS leads global efforts for Rare Disease Day, which it established in 2008. The observance is celebrated around the world on the last day of February each year and its purpose is to raise awareness of rare diseases as a public health issue. For more information about EURORDIS, visit www.eurordis.org.
Established in 1983, the National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. A nonprofit organization, NORD represents the 30 million Americans with rare diseases and is committed to the identification, treatment and cure of rare disorders through patient assistance, education, advocacy, research and patient/family services. For more information about NORD, visit www.rarediseases.org.
Retrophin is a fully integrated biopharmaceutical company dedicated to delivering life-changing therapies to people living with rare diseases who have few, if any, treatment options. The Company’s approach centers on its pipeline featuring late-stage assets targeting rare diseases with significant unmet medical needs, including sparsentan for focal segmental glomerulosclerosis (FSGS), a disorder characterized by progressive scarring of the kidney often leading to end-stage renal disease, and RE-024 for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood. Research exploring the potential of early-stage assets in several rare diseases is also underway. Retrophin’s R&D efforts are supported by revenues from the Company’s commercial products Thiola®, Cholbam® and Chenodal®.
i Global Genes Web Site. https://globalgenes.org/rare-diseases-facts-statistics/ Last accessed February 10, 2017
ii Rare Disease Day Web Site. http://www.rarediseaseday.org/article/what-is-a-rare-disease Last accessed February 10, 2017
iii EURODIS Web Site. http://www.eurordis.org/about-rare-diseases Last accessed February 10, 2017