Congenital Disorders of Glycosylation (CDG): common challenges and opportunities with other Rare Diseases

Congenital Disorders of Glycosylation (CDG)… If you have not heard of it, that is because it only affects about one in 20,000 people, which means that it is a Rare disease.
Like the majority of rare diseases, CDG are chronic serious genetic, life-altering and often life-threatening or fatal diseases due to multiple organ failure. The type and severity of problems associated with CDG vary widely among affected individuals, sometimes even among members of the same family . Walking, jumping, climbing ladders, running, reading, talking amongst other activities, prove to be difficult, often impossible in the majority of patients. The impact of this disorder goes beyond the physical manifestations of the disease. It includes economic burden, decreased productivity (both patient and caregivers), reduced social functioning, and lowered quality of life. In fact, despite the rapid advances in the field of Rare Diseases, fewer than 5% of rare diseases have drug therapies available . Most of rare diseases, like CDG, still have no treatment options at all. Taking the risk on a drug for these childrens and adults may not promise returns as high as common drugs. Additionally, scientists are making great progress each day, but more funding for CDG research needs to be given. Please learn more about this disease and major advances and opportunities in the field of rare diseases:

Share Button

Publicar un comentario

Puede usar las siguientes etiquetas de HTML:
<a href="" title=""> <abbr title=""> <acronym title=""> <b> <blockquote cite=""> <cite> <code> <del datetime=""> <em> <i> <q cite=""> <strike> <strong>