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Chiesi Global Rare Diseases Makes Waves at the WORLDSymposium 2025

Chiesi Global Rare Diseases, part of the Chiesi Group, is gearing up for an impactful presence at the 21st Annual WORLDSymposium™ Research Meeting from February 3-7, 2025, in vibrant San Diego, California. With a focus on advancing treatments for rare diseases, the company will present a total of 11 noteworthy abstracts exploring the depths of Fabry disease and alpha-mannosidosis.

A Deep Dive into Clinical Insights

The array of research includes long-term data from a decade-long study on velmanase alfa, particularly focusing on its efficacy for patients suffering from alpha-mannosidosis. Additionally, the presentations will shed light on patient tolerability while on treatment and embrace both patient and caregiver perspectives in effective disease management.

Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases, expressed enthusiasm for their upcoming presentation. He stated, "We are pleased to present a wide range of clinical data and insights related to the diagnosis, treatment, and appropriate monitoring of people living with Fabry disease and alpha-mannosidosis at the WORLDSymposium. As part of our commitment to improve patient outcomes in the lysosomal disease community, we continue our effort to learn, listen, and partner with patients and families."

Highlights of the Presentations

The poster presentations feature several key studies:

• - Pegunigalsidase alfa and Fabry Disease: A study by Dr. Nicola Longo compares infusion-related reactions in patients switching from agalsidase beta to pegunigalsidase alfa. Another study by Myrl D. Holida explores improved tolerability following the switch.
• - Clinical Assessment and Awareness: Other presentations seek to enhance awareness of Fabry disease among non-specialist healthcare providers and promote shared decision-making practices among patients and caregivers.
• - Long-term Findings in Alpha-Mannosidosis: Dr. Nathalie Guffon will discuss long-term motor function and quality of life in patients receiving velmanase alfa treatment over ten years, providing crucial insights for future therapies.

Satellite Symposia and Expert Insights

In addition to their numerous poster presentations, Chiesi is also hosting two satellite symposia during the WORLDSymposium to enrich the dialogue surrounding lysosomal diseases. The first symposium titled "Enzyme Replacement Therapy: Time to Act in Lysosomal Diseases" will focus on management strategies and treatment pathways for patients benefiting from enzyme replacement therapy. The second, titled "Red Light, Green Light – What About the Yellow Light?" will delve into treatment tolerability specific to Fabry disease, ensuring patients' and caregivers' voices are central in decision-making.

Commitment to Patients

Chiesi Global Rare Diseases has firmly established itself as an advocate for those affected by rare diseases, uniting dedicated efforts to enhance healthcare access and create therapies that improve quality of life. Their commitment goes beyond presenting data; it encompasses collaborating with the rare disease community globally to ensure patients receive the help they need.

With its forward-thinking approach to therapy and research, the Chiesi Group is setting the stage for a transformative dialogue at this year's WORLDSymposium. As the medical community gathers to share knowledge, Chiesi's contributions stand out, showcasing not only their dedication to research but also their passion for improving the lives of those impacted by rare diseases.