Summary of the Book The book is a story of a couple’s unrelenting search for a cure for a rare genetic disorder, the Pompe’s disease. It covers the struggles (and successes) of John and Aileen Crowley in their quest for a cure for this rare genetic disorder. The young family had a perfect family life. John had graduated from Harvard Business School, secured a well-paying job and bought a nice home for his family. The couple and their three children were living happily until the mother, Aileen, discovered that their fifteen-month-old daughter’s motor skills were deteriorating. Later, a diagnosis revealed that, the daughter, Megan, and her five-month-old brother, Patrick, had the Pompe’s disease, a rare genetic condition for which there was no effective cure. It was further revealed that the degenerative condition would affect the children’s muscles and lead to reduced ability to walk, talk or even eat. They were told that their two children would succumb to this disease before reaching puberty. In an attempt to save his children, John set out to search for a cure for the Pompe’s disease. He quit his job and launched a Biotech company, Priozyme Pharmaceuticals, which undertook research to identify a novel enzyme that would substitute the non-functional enzyme in the Pompe’s disease. However, the race for a causal treatment was a long and tedious one especially with regard to the clinical trials and the FDA approval process. Furthermore, competition among research scientists and pharmaceutical companies threatened to derail John’s search for the cure. In the final experiment, the top four drug compounds that had shown promising results were compared in a double-blind clinical trial and a potent compound was identified (Anand 67). A subsequent five-year drug development process yielded the cure, which was given to the children. The book essentially brings into light the lengthy drug development and approval process, and the competition among research teams, care providers, and pharmaceutical companies in the development of new drugs. Genetic and Clinical Aspects of the Pompe’s Disease Pompe’s Disease is an inherited rare genetic disorder that is estimated to affect one in every 40,000 children (Joppi, Bertele and Garattini 355). This degenerative disorder affects the skeletal and heart muscles, hence, a fatal condition. It has been found that mutations involving a gene that codes for alpha-glucosidase (GAA) enzyme cause the Pompe’s disease. The function of GAA in the body is to hydrolyze glycogen (sugar stored in cellular lysosomes) to release energy for use by the muscle cells. However, in the Pompe’s Disease, the GAA gene mutation encodes a defective GAA enzyme that lacks the ability to break down glycogen (Joppi, Bertele and Garattini 358). Consequently, the level of glycogen in the muscle cells increases leading to muscle degeneration. Alpha-glucosidase enzyme converts blood glucose into glycogen, which is reserve energy for muscle cells (Joppi, Bertele and Garattini 357). Glycogen degradation by alpha-glucosidase occurs in the lysosomes while the conversion of glucose to glycogen occurs in the cytoplasm, whereby insulin converts excess glucose into glycogen for lysosomal storage. The glycogen formed is then transported through a mechanism called ‘autophagy’ into the lysosomal cavity (Joppi, Bertele and Gara