Rare Diseases

We appreciate the impact that rare diseases have on individuals and families, and we are steadfast in our pursuit to listen, learn and understand their needs. We are driven by a purpose to improve lives through pharmaceutical innovation, bringing meaningful outcomes to the rare disease community, through strong partnership, innovative therapeutic solutions, rapid access, information and support.

Every patient’s story inspires us to deliver our best, blending compassion with expertise. We’re advocates, educators, innovators and supporters.

Our rare disease inspiration

ITALFARMACO’s inspiration for rare diseases is deeply rooted in the desire to leverage the company research and development (R&D) capabilities to address the significant unmet medical needs within this space. The company’s decision was galvanised by the challenges faced by families living with conditions like Duchenne muscular dystrophy (Duchenne), where treatment options are scarce and the impact on quality of life is profound.

Rare disease areas

Our current portfolio aims to address unmet medical needs in ALS and Duchenne.

Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic Lateral Sclerosis (ALS)

A progressive neurodegenerative disease that affects motor neurons and causes muscle weakness, paralysis and eventual respiratory failure.

Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD)

A genetic disorder that involves muscle degeneration, predominantly affecting boys, resulting in muscle weakness and a deficit or a loss of mobility.

Collaborating with the community

We are mindful of our responsibility and grateful for the opportunity to contribute positively to the lives of those affected by rare diseases. We foster a collaborative mindset that helps us listen, learn and adapt, enabling us to offer solutions and resources in a way that resonates with the needs of the rare disease community.

We Listen Rare Disease - Italfarmaco

We Listen

The voice of the patient is the guiding force in all our endeavours, resonating as a powerful reminder of why we do what we do. Our ears are always open to the rare disease community.

We Learn Rare Disease - Italfarmaco

We Learn

In our quest for knowledge, we blend our expertise with the needs of patients, Patient Advocacy Groups (PAGs), healthcare professionals, policy makers, regulatory agencies and industry partners, to forge a comprehensive understanding of rare diseases.

We Adapt Rare Disease - Italfarmaco

We Adapt

Our collaboration with the rare disease community reshapes our processes, utilising shared wisdom to align our approach with the genuine needs of patients, enhancing their care and support.

EU-ITFRD-24-00005.
Date of creation: June 2024