Mirum Pharmaceuticals Honors Alagille Syndrome Awareness Day with Launch of Disease Awareness Campaign
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment.
Children with ALGS live with severe and unrelenting symptoms that can impact their overall quality of life. Identifying these symptoms early on can lead to a diagnosis and an effective treatment plan.
“Many patients live for months or years before being diagnosed and some patients are misdiagnosed, leading to unnecessary and invasive surgeries that do not address the underlying cause of their liver disease,” said Chris Peetz, president and chief executive officer at Mirum. “Our objective is to provide a platform that helps healthcare providers understand the specific signs and symptoms of ALGS so they can be better equipped to identify and diagnose this rare disease and help their patients get the treatment they need.”
“This disease awareness campaign has the potential to help healthcare providers recognize Alagille syndrome in their patients in order to properly address this rare disease,” said Roberta Smith, president of the Alagille syndrome Alliance and parent of a child with ALGS. “The impacts of Alagille syndrome on families are many. We have only begun to scratch the surface in understanding disease burden and specifically, how different regions in the world are affected differently. As we celebrate International Alagille Syndrome Awareness Day, we will focus on sharing Alagille syndrome family stories that emphasize the critical need for continued research and treatment options for patients worldwide. The patient voice is the Alagille syndrome community’s greatest vehicle for change.”
Mirum’s goal is to advance the understanding and diagnosis of ALGS so that patients and their families will get the medical care and support they need to improve their quality of life. The company and its founders have been dedicated to understanding ALGS through the support of resources like the Global Alagille Alliance (GALA) for continued research, as well as genetic testing platforms that help effectively identify ALGS in patients. These resources assist in advancing research and the healthcare community’s understanding of this devastating rare disease.