Chiesi Global Rare Diseases today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases.
| - Real-world evidence provides valuable insights about the management and monitoring of patients with Fabry disease for healthcare providers, researchers, and industry partners - BOSTON, April 24, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases.
“There continues to be a general lack of awareness and a limited appreciation of the numerous unique challenges that rare disease patients face every day, including those who are living with Fabry disease,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases. “We are focused on putting patients at the center of everything we do, and we must understand the impact of Fabry disease on daily life from the patient’s perspective. The results of this survey will help us partner with healthcare providers to improve the care of patients with Fabry disease by addressing their most relevant unmet needs.” The 33-question online survey was designed to assess patient-reported patterns of disease monitoring, understand the patient’s perception of disease burden, and learn about the impact of therapy on patients’ quality of life. A total of 280 adult patients with Fabry disease in the U.S. and Canada responded, 84% of whom were currently receiving either enzyme replacement therapy (ERT) or chaperone therapy. Respondents reported that common symptoms included low energy or fatigue (72%), tingling (62%) or pain (60%) in the hands or feet, ringing in ears or hearing loss (54%), general body pains or pain crises (51%), and abdominal or stomach pain (50%). More than half (51%) of respondents reported their symptoms as bothersome (38%) or difficult to control (14%). In addition, temporary worsening of symptoms between infusions was reported by about half of respondents who were currently receiving or had previously received ERT, and less than half reported those worsening symptoms to their physician. Of those who reported worsening symptoms (n=59), 41% said that their physician prescribed medication to manage their symptoms or changed their treatment regimen. “Treatments for Fabry disease have improved the management and outcomes for patients, but important clinical and convenience limitations still exist,” said Lisa Berry, LGC, Division of Human Genetics at Cincinnati Children’s Hospital Medical Center, lead author of the publication. “Our analysis highlights the gap between current standard-of-care in disease monitoring and patient perception of disease progression among patients with Fabry disease.” About Chiesi Global Rare Diseases For more information visit www.chiesirarediseases.com. About Chiesi Group By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 31 countries, and counts more than 6,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For further information please visit www.chiesi.com. Chiesi Group Media Contacts Chiara Travagin Adam Daley PP-EF-0532 V1.0
SOURCE Chiesi Global Rare Diseases |
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