The genomics revolution over the past three decades has led to great strides in rare disease
(RD) research, which presents a major shift in global policy landscape. While RDs are …

Introduction: Rare diseases (RDs) are a severe, chronic, degenerative and often life-
threatening group of conditions affecting more than 30 million people in Europe. Their …

Background The availability of methotrexate and the introduction of multiple biological
agents have revolutionized the treatment of juvenile idiopathic arthritis (JIA). Several …

Although there is no single commonly accepted definition worldwide for rare diseases [1],
the European Commission has agreed that rare diseases are lifethreatening or chronically …

Objective The aim of this sco** review was to overview the cost-of-illness studies
conducted in rare diseases. Methods We searched papers published in English in PubMed …

Studies suggest a high economic burden among children and adolescents with
musculoskeletal pain. There is no summary in the literature on the overall economic burden …

Mental illness has been previously linked with autoimmune diseases, yet the associations
between parental mental illness and offspring's risk of autoimmune diseases is largely …

Objective Lack of medical and scientific knowledge on rare diseases (RD) often translates
into limited research on them and a subsequent lack of understanding of their economic …

Spinal muscular atrophy (SMA) is one of the most common severe hereditary diseases of
infancy and early childhood. The progression of this illness causes a high degree of …

Background Adolescents and young adults (AYA) with chronic somatic conditions have an
increased risk of comorbid depression and anxiety symptoms. Internet-and mobile-based …