Rare diseases are often ignored, and patients have little access to effective treatments. Once marketed, novel therapies developed through clinical trials are often expensive and their use is limited by payers claiming lack of sufficient data on the natural history of the disease and of benefit from these novel therapies.
Number of known rare diseases
Of rare diseases are genetic
Affected by rare diseases worldwide
As rare diseases involve small numbers, it makes it difficult to generate trial evidence. It is therefore necessary to make use of a collective effort to document the clinical expression of rare diseases and to ensure access to effective, safe, and affordable therapies.
SMASH (System and Molecular Approaches of Severe Hyperlipidemias) is an international network of top-level researchers, clinicians, patient organizations and stakeholders aimed at facilitating access to innovative treatments for rare and severe Lipid Disorders.
Ensure the proper development of this collaborative network to document rare dyslipidemias and provide patient access to emerging treatments in both developed countries and emerging economies.
We aim to improve the understanding of the issues, causes and consequences of rare dyslipidemias and promote access to personalized treatments for patients.
SMASH is committed to bridging the gap in healthcare disparities. We understand the challenges faced by patients with rare lipid diseases, especially those in remote areas or in regions with underdeveloped healthcare systems. Our mission is to ensure that effective treatment is not a privilege but a right accessible to all, regardless of geographic or economic barriers.
The rarity and geographical dispersion of patients with rare lipid diseases pose significant challenges in developing new therapies. SMASH’s network serves as a vital link, connecting these patients with the medical and research community. We focus on reducing development times for novel therapies by facilitating access to this crucial pool of patients, thereby accelerating the journey from research to treatment.
In the complex landscape of rare lipid diseases, shared knowledge is power. SMASH recognizes the critical need for healthcare professionals to have transnational access to shared data and information on similar diagnoses and cutting-edge research. We are dedicated to fostering collaboration and open exchange of information across borders, empowering professionals with the knowledge needed for groundbreaking advancements in treatment and care.
With the increasing pressure on health services and governmental institutions, the pace of developing innovative approaches for better accessibility and affordability of new treatments can be hindered. SMASH stands at the forefront, advocating for and driving the development of innovative solutions that can navigate and overcome these systemic challenges, ensuring that progress in the treatment of rare lipid diseases is not stalled by external pressures.
