Are you interested in learning about the latest trends and drug development in the rare disease field? Then you should check out a special Journalist Team Presentation from Global Data titled “Orphan Drugs: A Review of Key Development Opportunities.” This free webinar will look at possible future patterns and trends that may appear in the orphan drug field, and will focus on three distinct rare illnesses: mucopolysaccharidosis (MPS), transthyretin mediated amyloidosis (TTR amyloidosis), and atypical hemolytic uremic syndrome (aHUS).
About Mucopolysaccharidosis
Mucopolysaccharidosis is a group of metabolic disorders, also known as lysosomal storage diseases. There are several different types of this disease. In mucopolysaccharidosis, a certain enzyme which is responsible for the breakdown of glycosaminoglycan enzymes is either missing or dysfunctional. The different types of this disease are classified by which enzyme is affected. These diseases can cause severe, debilitating, progressive symptoms that may result in severe impairment and a severely shortened lifespan. To learn more about mucopolysaccharidosis, click here.
About TTR Amyloidosis
TTR amyloidosis is a disease in which abnormal, misfolded proteins called amyloid fibrils accumulate in body tissue. In this form of amyloidosis, the protein that is affected by the disease is called transthyretin. TTR amyloidosis is generally an inherited form of the disease in which a mutation causes the protein to misfold. Symptoms can include nervous damage, impaired movement, nausea, fatigue, chest pain, weight loss, and many others. A liver transplant is the best hope for a cure, but comes with many risks. To learn more about TTR amyloidosis, here.
About Atypical Hemolytic Uremic Syndrome
Atypical hemolytic uremic syndrome is a rare, progressive disease that is characterized by frequent and unregulated activation of the complement system, a component of the immune system that is instrumental in supporting the immune system’s role of destroying foreign agents in the body. This disease leads to the formation of small blood clots in many blood vessels throughout the body, which can be lethal without intervention. It is linked to a genetic mutation and has a high mortality rate, To learn more about atypical hemolytic uremic syndrome, click here.
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