“Rare Disease Day – GNE Myopathy” Beyond the Horizon: Innovations and Challenges in Rare Diseases

On February 21, 2025, at the headquarters of Tuscany Regional Committee, the Military Pharmaceutical Chemical Plant based in Florence, a production unit of the Agenzia Industrie Difesa (AID), in collaboration with UNIAMO, organized a meeting and training session on “GNE Myopathy”. During the event, the importance of research in the treatment of rare diseases was emphasized by Professor Maria Luisa Brandi from the FIRMO Foundation and Professor Silvia Priori from the IRCCS.

For over 20 years, the Military Chemical Pharmaceutical Plant has been supporting all individuals affected by rare diseases through the commitment of all personnel, both military and civilian, in the research, production, and importation of so-called “orphan drugs” and scarce drugs.

This activity is consolidated through close collaboration with the Ministry of Health, the Agenzia Italiana del Farmaco (AIFA) and the Istituto Superiore di Sanità (ISS).

The role of the Stabilimento Chimico Farmaceutico Militare has been recognized by its inclusion in the National Committee for Rare Diseases (CoNaMr).

The service provided to over 3,000 “rare” patients is also facilitated by the orphan drugs Portal created to improve access to treatment, the methods of obtaining and distributing the required drugs, and the Help Line service.

Source: AID

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