Recognizing the Voices and Journeys of Rare-Disease Families

Rare Disease Day, recognized each year on the last day in February, brings worldwide attention to the more than 300 million people living with rare conditions. For many families, the journey to diagnosis and treatment is long, complex, and filled with uncertainty. At Medicure, we believe these patients deserve visibility, understanding, and meaningful therapeutic options.

Our work in rare diseases - including the development of MC-1 for PNPO deficiency - reflects our commitment to advancing science where the need is greatest. Rare Disease Day is an opportunity to honor the resilience of patients and caregivers, recognize the dedication of clinicians and researchers, and reaffirm our mission to support underserved communities.

The Symbol of the Zebra and Rare Diseases

The zebra has become the emblem of the rare-disease community. In medical training, students are taught:

"When you hear hoofbeats, think horses, not zebras."

But for millions of people worldwide, the "zebra" is the diagnosis.

Rare Disease Day reminds us to look beyond the expected and recognize the patients whose conditions are often overlooked.

Why Rare Disease Day Matters

Over 7,000 rare diseases have been identified worldwide
Approximately 95% have no approved treatment
Many rare diseases disproportionately affect children
Families often face delayed diagnosis, limited resources, and few therapeutic options

Rare Disease Day raises awareness of these challenges and encourages collaboration across industry, advocacy groups, policymakers, and the scientific community

Medicure's Committment to Rare Diseases

Medicure is actively advancing research in rare pediatric neurometabolic disorders, including PNPO deficiency, an ultra-rare condition that can cause life-threatening neonatal seizures. Through our investigational therapy MC-1, we aim to bring forward a potential treatment option for families living with PNPO deficiency.

Our work is guided by three core principles:

1. Scientific Responsibility

We pursue rigorous research in areas where therapeutic options are scarce and the medical need is urgent.

2. Patient-Centered Focus

Families navigating rare diseases deserve clarity, compassion, and hope. Their experiences shape our priorities.

3. Long-Term Commitment

Rare-disease innovation requires persistence. We are committed to advancing therapies, supporting advocacy, and contributing to a stronger ecosystem for rare-disease research.

How You Can Support Rare-Disease Awareness

Wear stripes on Rare Disease Day
Share educational content on social media
Learn and amplify patient stories
Support organizations that fund research and provide family resources
Engage in conversations that help bring visibility to rare conditions

Every action - big or small - helps strengthen the rare-disease community.

Our Vision Moving Forward

Medicure is proud to stand with patients, caregivers, clinicians, and advocates on Rare Disease Day and every day. As we continue advancing MC-1 and exploring additional opportunities in rare diseases, we remain committed to improving outcomes for families who have waited too long for answers.

Together, we can build a future where rare diseases receive the attention, innovation, and support they deserve.

Recognizing the Voices and Journeys of Rare-Disease Families

The Symbol of the Zebra and Rare Diseases

Why Rare Disease Day Matters

Medicure's Committment to Rare Diseases

1. Scientific Responsibility

2. Patient-Centered Focus

3. Long-Term Commitment

How You Can Support Rare-Disease Awareness

Our Vision Moving Forward

Medicure Celebrates Rare Disease Day

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