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.
Expanding Access to Rare Disease Treatments
In 2024, Norameda introduced four new treatments for rare diseases, marking a significant milestone in improving patient access to essential therapies. The newly launched medicines address:
- Alpha mannosidosis – a rare genetic disorder affecting lysosomal function
- Fabry disease – a metabolic disorder caused by enzyme deficiency
- Leber’s hereditary optic neuropathy (LHON) – a mitochondrial disease leading to vision loss
- Narcolepsy – a neurological condition that affects sleep regulation
A key achievement in 2024 was the launch of a new treatment for Fabry disease and the successful representation of Wakix for narcolepsy treatment. These efforts demonstrate Norameda’s commitment to ensuring that patients receive timely and effective treatment options.
Lithuania: A Leader in Alpha Mannosidosis Diagnosis
One of the notable successes in Lithuania’s rare disease landscape is the diagnosis of Alpha mannosidosis. With seven diagnosed cases, Lithuania has exceeded the number of cases identified in the Scandinavian region, highlighting 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 their quality of life. Lithuania’s achievements in Alpha mannosidosis diagnosis showcase the effectiveness of collaboration between healthcare institutions, diagnostic centers, and pharmaceutical partners.
The Complexity of Rare Disease Care Pathways
Managing rare diseases requires specialized knowledge, multidisciplinary collaboration, and coordinated patient care pathways. The complexity of these conditions means that standard treatment approaches are often insufficient.
Recognizing this challenge, the European Union established 24 European Reference Networks (ERNs) in 2017 to facilitate cross-border cooperation in rare disease treatment. The Vilnius Santaros Clinic has actively participated in these networks, contributing to knowledge-sharing and best practices in rare disease management.
Norameda plays a crucial role in streamlining care pathways, ensuring that patients receive integrated and high-quality treatment. By fostering collaboration between healthcare professionals, patient advocacy groups, and research institutions, Norameda aims to improve outcomes for individuals affected by rare diseases.
Driving Innovation and Knowledge Exchange
As a leading pharmaceutical company, Norameda is dedicated to advancing medical innovation and fostering international collaboration. By supporting the Rare Diseases Coordination Centre at Vilnius Santaros Clinic, Norameda contributes to groundbreaking research, educational initiatives, and improved patient access to life-changing treatments.
The company’s long-term vision includes:
- Expanding treatment options for rare diseases
- Enhancing diagnostic capabilities through strategic partnerships
- Promoting cross-border collaboration to share best practices in patient care
A Future of Better Care for Rare Disease Patients
The journey toward better rare disease treatment requires continuous innovation, strong partnerships, and a commitment to patient well-being. Norameda remains at the forefront of this mission, ensuring that patients with rare conditions receive the care they deserve.
By facilitating access to new therapies, supporting diagnostic advancements, and moderating knowledge exchange between countries, Norameda is shaping the future of rare disease management in Lithuania and beyond.
Together, we can make a lasting impact.