Chiesi Global Rare Diseases to Present at the 19th Annual WORLDSymposium™ Research Meeting

- Eight abstracts include long-term results from Phase 3 BALANCE and BRIGHT studies in Fabry disease and insights from the SPARKLE registry in alpha-mannosidosis -

BOSTON, Feb. 15, 2023 /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 that eight abstracts have been accepted for presentation at the 19^th Annual WORLDSymposium^™ Research Meeting to be held February 22–26, 2023 in Orlando. The company will also support satellite symposia focused on lysosomal diseases including Fabry disease and alpha-mannosidosis.

Two oral presentations during the "Clinical Applications" session on Friday, February 24^th beginning at 1pm EST will provide updates from the company's clinical development program in Fabry disease.

• Eric Wallace, M.D., Co-Director of the University of Alabama at Birmingham Fabry Disease Clinic, will present an abstract titled, "First results of a head-to-head trial of pegunigalsidase alfa vs. agalsidase beta in Fabry disease: 2-year results of the phase 3 randomized, double-blind, BALANCE study."
• John Bernat, M.D., Ph.D., Medical Director of the Iowa Lysosomal Storage Disorders Center, will present an abstract titled, "Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study."

The company is announcing the presentation of six additional posters during WORLDSymposium:

• "Patient-reported experiences with Fabry disease monitoring and disease burden in the real-world setting: results from a double-blind, cross-sectional survey" - Lisa Berry, LGC, Division of Human Genetics at Cincinnati Children's Hospital Medical Center
• "Safety and compliance of home infusion of velmanase alfa for the treatment of alpha-mannosidosis in the clinical trial and real-world settings" - Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France
• "Baseline characteristics of a real-world population with alpha-mannosidosis: insights from the SPARKLE registry" - Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France
• "Real-world clinical profiles of patients with alpha-mannosidosis: baseline evaluations from the SPARKLE registry" - Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany
• "Systematic Literature Review on the Epidemiology, Diagnosis, and Natural History of Alpha Mannosidosis" - Patricia Dorling, Head of Global HEOR at Chiesi USA, Inc.
• "Systematic Literature Review on the Burden of Illness, Health-related Quality of Life, and Treatment Options in Pediatric and Adult Patients with Alpha Mannosidosis" - Patricia Dorling, Head of Global HEOR at Chiesi USA, Inc.

The company will also support two satellite symposia during WORLDSymposium:

• On Friday, February 24^th beginning at 5:15pm EST, Robert Hopkin, M.D., Clinical Geneticist at Cincinnati Children's Hospital Medical Center, will serve as Chair of a symposium titled, "One size does not fit all: Monitoring and management of anti-drug antibodies in lysosomal diseases with a focus on Fabry disease." Additional speakers will include Derralynn Hughes, M.D., Professor of Experimental Haematology at the University College London, and Maria João Gonçalves, M.D., Instituto de Medicina Molecular.
• On Saturday, February 25^th beginning at 11:45am EST, Barbara Burton, M.D., Professor of Pediatrics at Lurie Children's Hospital of Chicago, will serve as Chair of a symposium titled, "Pinpointing Alpha-Mannosidosis on the Map: Insights from the diagnostic odyssey of patients." Additional speakers will include Nicole Muschol, M.D., University of Hamburg, Karolina Stępień, M.D., Salford Royal NHS Foundation Trust, and Florian Lagler, M.D., Paracelsus Medical University Salzburg.

About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit www.chiesirarediseases.com.

About Chiesi Group
Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment.

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 at becoming net-zero by 2035.

With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 30 countries, and counts more than 6,000 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 Global Rare Diseases Media Contact
Adam Daley
Berry & Company Public Relations
1-212-253-8881
[email protected]

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