Amicus Therapeutics to Showcase Research at Global Muscle Congress

Amicus Therapeutics to Present at World Muscle Society Annual Congress

Amicus Therapeutics (NASDAQ: FOLD) is set to participate in the prestigious World Muscle Society's Annual Congress, where it will showcase pivotal research on treatments for Pompe disease. This event highlights the company's dedication to addressing rare diseases through innovative research and breakthroughs.

Key Highlights from the Congress

During the congress, Amicus Therapeutics will present three essential posters that delve into its cutting-edge development programs for Pompe disease. Each poster session promises to deliver critical insights and findings that enhance our understanding of this condition.

Poster Display: Focus on Pompe Disease

The first noteworthy abstract tackles the efficacy of Cipaglucosidase alfa + miglustat in late-onset Pompe disease, emphasizing a shift in treatment strategies for two non-ambulatory patients switching from conventional therapies. This session is set for Friday, October 11, during the conference, making it a key highlight for attendees.

Engagement with Leading Experts

Renowned researcher Barry J. Byrne, MD, PhD from the University of Florida will lead this presentation, providing invaluable insights from clinical experiences. Conference participants will have the opportunity to explore the latest updates in Pompe disease treatment methodologies and outcomes.

Further Insights and Presentations

The second poster, titled Post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease, will also be showcased in the same session. This work, presented by Dr. Mark Roberts from Salford Royal NHS Foundation Trust, is vital for understanding patient outcomes in real-world settings.

Pioneering Research

Moreover, the congress will feature a presentation on Miglustat: a first-in-class enzyme stabilizer for late-onset Pompe disease, delivered by Dr. Tahseen Mozaffar from the University of California, Irvine. This presentation aims to shed light on new therapeutic approaches that hold promise for patients suffering from this challenging condition.

Commitment to Rare Diseases

Amicus Therapeutics is more than just a biotechnology company; it is a dedicated ally for individuals afflicted with rare diseases. The company’s focus spans from drug discovery to advanced development, with a commitment to providing high-quality medical solutions that meet the unique needs of patients across the globe.

Future Perspectives and Vision

Through its active engagement in scientific forums like the World Muscle Society Annual Congress, Amicus Therapeutics continuously seeks to improve and expand its pipeline of innovative medicines. With a patient-centric approach, the company is well-positioned to reshape the future of treatment for rare diseases.

Frequently Asked Questions

What will Amicus Therapeutics present at the congress?

Amicus will present three posters focused on Pompe disease, highlighting recent research and treatment advancements.

Who will lead the presentations at the congress?

Notable presenters include Dr. Barry J. Byrne, Dr. Mark Roberts, and Dr. Tahseen Mozaffar, leading experts in the field.

When is the World Muscle Society Annual Congress held?

The congress takes place from October 8-12, 2024, providing a platform for sharing the latest in muscle disease research.

What is the primary focus of Amicus Therapeutics?

Amicus is focused on developing novel therapies for rare diseases, especially those that impact patient health significantly.

How can I learn more about Amicus Therapeutics?

For additional information, visit Amicus Therapeutics' website and follow their updates on social media platforms like X and LinkedIn.

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