and Rare Genetic Diseases
are our fields of expertise.
Rare diseases affect a very small percentage of each population in a country, but most are associated with life-long disability, often starting in childhood. Many of these rare diseases are chronic, degenerative and potentially life-threatening conditions, often under-diagnosed or diagnosed too late. A supportive, dedicated framework has been set up in the USA, EMA and many others geographies to promote a greater understanding of these diseases, stimulate more public and private research and to encourage innovative biopharma companies to develop and make available breakthrough treatments for some of these diseases.
Access to these products can be complex, challenging and costly. For over 15 years, Mawdsleys has been the reference partner for many physicians and their patients to source the products patients need as quickly as possible whilst ensuring compliance with regulatory systems. With its well-established Unlicensed Medicines organisation in more than 52 countries (including Europe, Brazil, Israel), Mawdsleys Specialty Pharma has the expertise and resource to source niche medicines that are not licensed or readily available for the patients suffering from these rare diseases. In many cases, Mawdsleys Specialty Pharma is also able to help patients access funding for these important but costly medicines.