Actelion Honored for Its Research Work and Support for People with PAH and Other Rare Diseases

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Actelion honored

Actelion Ltd. was recently recognized for its work in the treatment of rare diseases with the the EURORDIS Company Award 2016, an honor given by the European Organization for Rare Diseases (EURORDIS). The award, given to businesses for pioneering work in drug discovery and development, also acknowledged Actelion’s work with pulmonary arterial hypertension (PAH) patient groups, and its support for PAH patients and caregivers.

Actelion’s efforts include raising awareness regarding rare diseases and the challenges faced by patients through the development of educational resources such as the PAHuman eBook, detailing the nature of PAH was a chronic and life-threatening disorder and its impact on patients’ lives. Other initiatives include the “Think Again. Think NP-C” campaign, encouraging earlier diagnosis of Niemann-Pick type C disease, a rare and neurodegenerative condition whose diagnosis is rather difficult due to the rarity and heterogeneity of its symptoms.

“Actelion has provided invaluable support to our activities and, as a result, our collaboration has led to many great achievements over the years. It’s clear that Actelion’s commitment extends beyond the discovery and development of new medicines and includes initiatives that increase knowledge and understanding of PAH and other rare diseases, which is crucial to organizations like ours,” Gerald Fischer, CEO of the European Pulmonary Hypertension Association (PHA Europe), said in a press release.

Over 65,000 patients with rare diseases were benefiting from treatments with drugs developed by Actelion by the end of 2015, the company said in its release.

“I am very proud that, together with the PAH community, Actelion has brought about not one, but several paradigm shifts in PAH care. The three established pathways in PAH are now well served, but our discovery efforts continue. We are turning to new pathways in PAH, helped by our strong connections with academia, to further improve the prognosis for patients living with this devastating disease. Actelion also plays a leadership role in Niemann-Pick type C disease, where the lives of many patients have been positively impacted. I am convinced that with our strong and innovative research, Actelion will continue to advance care in many rare diseases,” said Martine Clozel, MD, chief scientific officer at Actelion.