
Norameda’s Commitment to Advancing Rare Disease Treatment
Rare diseases present unique challenges in diagnosis, treatment, and patient care. As a company dedicated to improving healthcare, Norameda continues to prioritize rare diseases by expanding its portfolio of innovative medicines and strengthening partnerships with leading medical institutions.
Improving Access to Rare Disease Treatment
In 2024, Norameda introduced four new treatments for rare diseases, significantly improving patient access to life-saving therapies. The latest medicines target:
Alpha-mannosidosis – a rare genetic disorder affecting lysosomal function
Fabry disease – a metabolic disorder caused by enzyme deficiency
Leber hereditary optic neuropathy (LHON) – a mitochondrial disease causing vision loss
Narcolepsy – a neurological condition affecting sleep regulation
One of the most significant achievements of 2024 was the introduction of a new treatment for Fabry disease and the successful representation of Wakix for narcolepsy treatment. These milestones reaffirm Norameda’s commitment to ensuring that patients receive timely and effective treatment solutions.
Lithuania – A Leader in Alpha-Mannosidosis Diagnosis
One of Lithuania’s most remarkable achievements in the field of rare diseases is the diagnosis of Alpha-mannosidosis. Seven cases of the disease have been registered in Lithuania — more than in the entire Scandinavian region combined. This demonstrates the country’s progress in rare disease diagnostics.
Early and accurate diagnosis is crucial for patients with rare diseases, as it enables timely interventions that can significantly improve quality of life. Lithuania’s achievements in this area highlight the successful collaboration between healthcare institutions, diagnostic centers, and pharmaceutical partners.
The Complexity of Rare Disease Treatment
Managing rare diseases requires specialized expertise, interdisciplinary collaboration, and coordinated patient care pathways. Due to the complexity of these conditions, conventional treatment approaches are often insufficient.
Recognizing this challenge, the European Union established 24 European Reference Networks (ERNs) in 2017 to promote cross-border collaboration in rare disease care. Vilnius Santaros Clinics actively participate in these networks, contributing to knowledge exchange and the dissemination of best practices in rare disease management.
Norameda plays an important role in optimizing patient care processes, ensuring that patients receive integrated and high-quality treatment. By fostering collaboration among healthcare professionals, patient organizations, and research institutions, Norameda aims to improve the quality of life of people living with rare diseases.
Driving Innovation and Knowledge Sharing
As a leading pharmaceutical company, Norameda is committed to promoting medical innovation and international collaboration. By supporting the Rare Diseases Coordination Center at Vilnius Santaros Clinics, the company contributes to advanced research, educational initiatives, and improved patient access to vital treatments.
Norameda’s long-term vision includes:
Expanding treatment options for rare diseases
Strengthening diagnostic capabilities through strategic partnerships
Promoting cross-border collaboration to share best practices in patient care
Towards a Better Future for Rare Disease Patients
The path toward better rare disease treatment requires continuous innovation, strong partnerships, and a genuine commitment to patient well-being. Norameda remains at the forefront of this mission, ensuring that patients living with rare diseases receive the care they need.
By expanding access to new therapies, advancing diagnostic capabilities, and facilitating international knowledge exchange, Norameda is shaping the future of rare disease management in Lithuania and beyond.
Together, we can create a lasting impact.