IRCCS Ospedale Galeazzi–Sant’Ambrogio recognized as a regional reference center for the treatment of amyloidosis

Resolution 5056 is part of the update to the Rare Disease Network outlined in the National Rare Disease Plan 2023–2026 and Law 175/2021. The provision establishes new criteria for selecting and verifying reference centers, requiring:

• proven clinical expertise
• advanced technological equipment
• a multidisciplinary team
• the ability to ensure continuous patient care

In this framework, Galeazzi–Sant’Ambrogio demonstrated full compliance with all requirements, including:

• a dedicated clinic for myocardial and pericardial diseases
• state-of-the-art diagnostic technologies (advanced echocardiography, cardiac MRI, multimodality imaging)
• a multidisciplinary team with specialized expertise in cardiac amyloidosis

As Prof. Andreini emphasizes: “We have structured a dedicated clinical pathway that guarantees continuity of care and quality standards aligned with international guidelines.”

Transthyretin amyloidosis (ATTR) is a deposition disease caused by the abnormal accumulation of transthyretin, a protein produced by the liver that, in its pathological forms, misfolds and deposits in tissues, particularly the myocardium.

This process leads to myocardial thickening and loss of elasticity, progressively resulting in restrictive cardiomyopathy and refractory heart failure.

The disease primarily affects older adults and, if not diagnosed promptly, can progress rapidly and severely. Early recognition is therefore essential to initiate targeted therapies and significantly improve patient outcomes.

Patients with TTR amyloidosis treated at IRCCS Galeazzi–Sant’Ambrogio can benefit from innovative disease-modifying drugs capable of stabilizing the transthyretin molecule and preventing its transformation into the amyloidogenic form that accumulates in tissues.

These treatments, approved by AIFA, represent a major change compared with traditional management, which focused exclusively on symptomatic therapy.

“These drugs are a true step forward. By acting on the molecular mechanism of the disease, they slow its progression and improve patients' quality of life. Our center will handle not only prescription, but also clinical and instrumental follow-up, ensuring continuous monitoring of therapeutic efficacy and safety,” notes Prof. Andreini.

Recognition as a prescribing center allows the University Cardiology Unit of IRCCS Ospedale Galeazzi–Sant’Ambrogio to manage the entire therapeutic process through personalized treatment plans, scheduled follow-up visits, and regular monitoring.

All services are provided within the National Health Service, with no additional costs for patients.

The Hospital, a national reference point for cardiology and myocardial diseases, thus reaffirms its mission: combining research, innovation, and clinical excellence to improve patients’ lives.