Orphazyme recognizes Rare Disease Day 2018

Orphazyme A/S
Investor news                                                                                                                                     
No. 03/2018                                                                                                                                        
                                                                                                                                                           

Copenhagen, February 28, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases today announced its support for Rare Disease Day. Observed annually on the last day of February, Rare Disease Day is dedicated to raise awareness about the public health issues associated with rare diseases. This is the 10^th anniversary of Rare Disease Day, which was established by the European Organisation for Rare Diseases (EURORDIS), an organization representing rare disease patients in Europe, and the day is coordinated in partnership with the National Organization of Rare Disorders (NORD) in the US, with now more than 70 nations actively participating.

"We join hands with patients, family members, healthcare professionals, researchers, and government agencies worldwide to call attention to the unique challenges patients and their families with rare diseases face,” says Anders Hinsby, Chief Executive Officer of Orphazyme. "It is our mission to advance research and develop therapies that will improve care for people with rare diseases.”

The European Union considers a disease as being rare if it affects less than 1 in 2,000 citizens, and rare diseases (also known as "orphan” diseases) are defined in the USA as affects fewer than 200,000 citizens. Given their rarity, these conditions are often difficult to diagnose and treat. Patients can endure a long odyssey involving many specialty doctors, tests, and numerous misdiagnoses. According to a report by Global Genes, it takes on average of 7 physician visits and nearly 5 years from the onset of symptoms to obtain an accurate diagnosis for those affected with a rare disease.

In honor of this year’s Rare Disease Day theme, "Show Your Rare, Show You Care,” Orphazyme will host Toni Mathieson, Chief Executive Officer in Niemann-Pick UK and Executive Secretary of the International Niemann-Pick Disease Alliance, to share her journey as a parent with children with Niemann-Pick disease Type C and as a leader in the rare disease community.

"Rare Disease Day provides an important opportunity to raise visibility of these little-known conditions and the devastating impact they can have on patients and their families. Through the events being held today, we want to amplify the voices of those affected by rare diseases and highlight the need for effective research and timely access to expert care and treatment” says Toni Mathieson.

For more information about Rare Disease Day activities worldwide, visit www.rarediseaseday.org. Also, go to our Twitter account and LinkedIn page to get a glimpse of the Orphazyme employees participating in activities to recognize Rare Disease Day 2018.

About Orphazyme A/S
Orphazyme is a Danish biopharmaceutical company focused on the development of treatments for patients living with life-threatening or debilitating rare diseases. Our research is concentrated on the cell-protective properties of the body’s own rescue system for diseases caused by misfolded proteins or lysosomal dysfunction. The company’s lead candidate, arimoclomol, is in development for four severe orphan diseases; two muscle-wasting diseases, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS), and two lysosomal storage diseases, Niemann-Pick disease Type C (NPC) and Gaucher disease. We are listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit?www.orphazyme.com. 

03-2018 Orphazyme recognizes Rare Disease Day 2018