The cure - Book Report/Review Example

This occasion motivated the two to dedicate their time for the disease since meeting in 2001 in New Jersey (Anand, 2010). The fact that the disease had no known cure even accentuated John to pursue biotechnology and eventually became a successful specialist in the field. This is not to say that the book is meant to demonstrate how successful someone can be or to thrill on John’s success, the book proceeds in enumerating the several challenges that Crawleys faced in his pursuit for the course- these were specifically in the protocols involved in the clinical processes.

He also faced challenges of the rivalries that existed between the medical procedures as well as those of pharma firms. Later, John made a keen follow up of his case regarding the disease following its publication in the wall street journal (Anand, 2010). In the book, the cure, it is also recounted how John depended on flouted regulations, quick money schemes and bucked the medical establishment in his effort to raise the $ 100 million. He determination of Crowleys as demonstrated in this book will raise the eyebrows of the entrepreneurs in noting the efforts while to some people it will be a sobbing moment in retrospect of their plight.

The only part of the book that reduces it meaning and makes it to be construed to be another film directed by Ford is the cover plurb. Genetic and Clinical Aspects of Pompe Disease Pompe is described to be inherited and consist of accumulation of glycogen in the body-this is a complex sugar. When this glycogen accumulates in the body, they do impair the ability of the tissues, organs and muscles to operate (Anand, 2010). Pompe has been described in three types, the classic infantile-onset, non-classic infantile-onset, and the late-onset, this is based on age.

The classic infantile-onset is mostly found in infants and leads to death after a year if not attended to; it causes hepatomegaly, myopathy, and hypotonia leading to death in the infants (Anand, 2010). The non-classic do appears after the age of one; it is characterized by cardiomegaly and delayed motor nerves. Those affected are also prone to heart failure leading to breathing problems and death follows. The late onset is normally experienced in the adolescent, compared to the two others; it is much milder than the two.

It is also characterized by weakening of the muscles especially those of the legs and the trunk. The muscles impairment may also lead to breathing difficulties that may occasion respiratory problems. Concept of “Orphan “Drug This concept relates to those pharmaceutical agents that have been developed for treating a very rare medical condition with the condition also being referred to as orphan disease. In normal situation, orphan drugs do receive funding incentives for the encouragement of the drug discovery, this is because without this support, the results may not be achieved or may be delayed because so the economics involved in the discovery of the drugs (Anand, 2010).

The orphan status of the drug is always given as matter of public policy and has always culminated into medical breakthrough. Just like the conventional drugs, Orphan drugs are also subjected to the rigor of the regulation and testing in order to ascertain their efficacy. Roles of Pharmaceutical companies and FDA in Drug development It will be understood that the drugs we have in our cabinets have travelled a long path for