RATIONALE

ALPHA MANNOSIDOSIS AND MUCOPOLYSACCHARIDOSIS: OPTIMIZING PATIENT JOURNEYS

Alpha Mannosidosis and Mucopolysaccharidoses (MPS) present a broad and heterogeneous clinical spectrum that often challenges timely diagnosis and effective care coordination. The non-specific and overlapping nature of their symptoms with other disorders contributes to diagnostic delays and under-recognition, particularly in healthcare systems lacking dedicated tools or structured pathways.

Given the progressive course and multisystem involvement of these diseases, a coordinated, multidisciplinary approach is essential to ensure early intervention and sustained management. Advances in newborn screening, digital health technologies, and artificial intelligence offer promising avenues to support earlier identification and more accurate patient stratification..

This international educational event brings together experts from diverse specialties to share real-world experiences, discuss evolving therapeutic strategies, and highlight the importance of PROMs in evaluating outcomes and shaping access to care. By fostering clinical collaboration and awareness, the program aims to enhance the quality, continuity, and equity of care for patients affected by rare lysosomal storage disorders..

Learning Objectives

Recognize key clinical features of Alpha Mannosidosis and Mucopolysaccharidoses (MPS I, II, IVA, IVB) to support early and differential diagnosis

• Understand diagnostic challenges in Lysosomal Storage Disorders and explore available tools and strategies to reduce diagnostic delay, including AI and newborn screening programs.

• Evaluate current therapeutic options, including long-term data on Velmanase Alfa, and discuss unmet needs in the treatment of skeletal complications.

• Promote a multidisciplinary approach in patient care through interactive case simulations and cross-specialty collaboration.

• Appreciate the role of patient-reported outcomes (PROMs) in assessing therapeutic impact and guiding reimbursement decisions

Target Audience

• Pediatricians
• Clinical Geneticists
• Diabetologists
• Neurologists
• Internal Medicine Physicians
• Orthopedic Surgeons
• Ophthalmologists
• Otolaryngologists
• Hematologists
• Nephrologists
• Clinicians specialized in Hereditary Metabolic Diseases

PROGRAMME

Session 1 –  THE PATH TO DIAGNOSIS
Moderator: M. Magner

Session 2 – ESTABLISHED TREATMENTS AND THEIR CAVEATS
Moderator: F. Lagler

Session 3 – THE MEANING OF MANAGEMENT
Moderator: F. Lagler

Session 4 – NAVIGATING COMPLEX CLINICAL CASE
Moderator: F. Lagler

Session 5 – RARE AND AWARE
Moderator: D. Zafeiriou

SCIENTIFIC COORDINATOR

Florian Lagler (Austria)

Clinical Research Center Salzburg GmbH
Medical Simulation Center Salzburg

Conference venue

Imperial Riding School Autograph Collection
Ungargasse 60, 1030 Wien, Austria | Vienna

REGISTRATION

For any information, please contact:
First Class srl – Milan – Via Vittoria Colonna, 40 – 20149,Milan – Italy
E-mail: valeria.ballo@fclassevents.com

ACCREDITATION

The programme will be submitted for CME accreditation to the European Accreditation Council for Continuing Medical Education (EACCME®).

TRAILS 2025 – 4th Edition

Alpha Mannosidosis and Mucopolysaccharidosis: optimizing patient journeys

Vienna, October 28th-29th 2025

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