European Organization for Rare Diseases

immoral business tactic used by pharmaceutical companies to generate large revenues involves the slight modification of an existing compound and using the new compound in the treatment of an orphan disease. In 2009 the company BioMarin Pharmaceutical created the drug Firdapse. The active substance is amifampridine and is used to treat the orphan disease, Lambert-Eaton myasthenic syndrome. In 2012 it was revealed that the product was simply a slight modification of 3,4 diaminopyridine, an inexpensive compound that had been around for a few decades. Firdapse was being sold for about 60 times higher compared to 3,4 diaminopyridine. [12] So what can be done to solve these dilemmas that orphan drugs face? Should we allocate more resources into funding orphan drug research, and if so how much? Allowing for greater incentives to pharmaceutical companies could certainly help lower prices and also increase a pharmaceutical company’s interest in developing drugs for rare diseases; however, this may be unfavorable due to the fact that a burden would most likely be put on society as a whole thru an increase in taxes. Today there are many patients who utilize Patient-Assistance Programs as a resource for for making treatments more accessible and affordable. NORD (National Organization for Rare Disorders) has established programs that aids patients who are insured by lowering their premiums and coypayments. They currently have 34 of these Patient-Assistance programs. [11] Programs such as…

company to gain share in the global market while keeping its core values unchanged. The focus of the company from the beginning has been putting patients first and the focus on the “two-price policy” – full price or free for patients in need and who could not afford it. HAND was created to help an untapped market, where neglected diseases such as tuberculosis kill as much as 2 million people a year in…

The most recent diagnostic criteria by Cohen et al. includes a number of specific benign tumors and malformations common with the condition. Some key specific criteria include cerebriform connective tissue nevi, hemihyperplasia with bone deformity , and dysregulated adipose and vascular tissue (Figure 3). The tumors caused by the disease are largely benign, and include lipomas, ovarian cystadenomas, monomorphic parotid adenomas , and meningiomas, though many other tumors benign and malignant…

Angelman Syndrome Description of the Disease An extremely rare and uncommon disease known as Angelman Syndrome is a very complex neurodevelopmental and monogenic disorder (Bailus, 2014). The term neurodevelopmental refers to the shape, reshape, state and generation of the nervous system during the embryonic stages of life, while the term monogenic can be explained as a disease, which is controlled and inherited, by a single pair of genes (Gentile, 2009). It is estimated that Angelman Syndrome…

Amicus Therapeutics is a biopharmaceutical company based in Cranbury, New Jersey. The company’s focus is on finding advanced therapies and treatments for a broad range of human genetic diseases. These diseases, known as orphan diseases, typically affect fewer than 200,000 people nationwide. Currently the company’s main concentration is on treatments for Lysosomal Storage Disorders such as Fabry disease and Pompe disease. Amicus Therapeutics has directed their development on the…

conditions that are perceived as solvable whereas conditions are external events that we encounter in our everyday lives. It is important for political actors to correctly identify problems and their causes so that the existing understandings of an issue can be challenged. For instance in the Medicare Catastrophic Coverage case, gaps in Medicare coverage left elderly Americans vulnerable to catastrophic illness. However, political actors realized the flaws in Medicare coverage and decided…

slave labor. Interactions between the natives, slaves, and Europeans were also different. In Latin America, many men had relations with native and slave women, eventually some married the natives. This caused a new social order to develop between the people in Latin American colonies, dividing everyone based on race, with titles such as mestizo, mulattoes, and zambos. In North American colonies, such relationships were frowned upon and were rare, but some mixing of the cultures occurred. The…

Vaccines are a biological preparation that provides active acquired immunity to a particular disease. When vaccines are given, it is an injection of a muted down version of a particular disease that allows the body to naturally become immune. Vaccinations against a variety of diseases such as polio, measles, pertussis, rubella and hepatitis B have been successful in preventing negative health effects and death. Opponents of making vaccinations mandatory argue vaccines are not safe and cause…

compared to the financial tolls of most diseases. Also, immunizations not only protect a single child, but those around as well. Being informed is the number one way to make educated decisions on vaccines. Jumping to conclusions can be dangerous; however, research based proven facts are reliable and safe (Centers for Disease Control and Prevention).…

Individuals who lived before 1500 often spent their entire lives in the same place. This was due to the fact of very little infrastructure for transportation and there usually wasn’t a reason to leave home. The poor would often travel to nearby marketplaces for business and trade. However, this was still very small in terms of travel. Although travel before 1500 was rare in Europe, most individuals were aware of traveling and the immediate world around…