Extraordinary Measures - Pompe Disease (based in a true story)

[Marina9]

Yesterday at night I came across this movie. I had no idea about the disease called Pompe. First of all I think the will and courage the dad and mom had to fight for the life of his daughter and son was just amazing. Of course as a parent the love you have for ur children goes beyond explanation, but nowadays I think that with all these diseases, and how the conventional doctors act towards them, a lot of people loose hope and doctors are just part of a business and not truly caring about the people.

Don't want to spill the whole movie, but the dad and mom find a doctor who is doing research on Pompe disease and how to help patients, but he has no money, so when Crowley (the dad) finds Stonehill (the doctor) they come together and create a whole plan to raise money and carry out Stonehill's theory for the treatment.

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The movie was based on a book called "The Cure:"
https://www.theguardian.com/books/2010/feb/12/pharmaceuticals-industry

The new Harrison Ford film Extraordinary Measures, released in the UK on February 26 2010 was inspired by The Cure, written by Geeta Anand, a Mumbai-based journalist and Pulitzer Prize winner. The book is based on the true story of John and Aileen Crowley's quest to develop a treatment for their two children, who suffer from Pompe disease – a rare and often fatal muscular disease that affects the heart, skeletal muscles, liver and nervous system. John Crowley started his own biotech company Novazyme, which eventually helped to discover a revolutionary new treatment. Anand describes the journey she took in writing the book and seeing its transformation from page to screen

And then I was doing a little bit of research on the disease:

Pompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally.

Researchers have described three types of Pompe disease, which differ in severity and the age at which they appear. These types are known as classic infantile-onset, non-classic infantile-onset, and late-onset.

The classic form of infantile-onset Pompe disease begins within a few months of birth. Infants with this disorder typically experience muscle weakness (myopathy), poor muscle tone (hypotonia), an enlarged liver (hepatomegaly), and heart defects. Affected infants may also fail to gain weight and grow at the expected rate (failure to thrive) and have breathing problems. If untreated, this form of Pompe disease leads to death from heart failure in the first year of life.

The non-classic form of infantile-onset Pompe disease usually appears by age 1. It is characterized by delayed motor skills (such as rolling over and sitting) and progressive muscle weakness. The heart may be abnormally large (cardiomegaly), but affected individuals usually do not experience heart failure. The muscle weakness in this disorder leads to serious breathing problems, and most children with non-classic infantile-onset Pompe disease live only into early childhood.

The late-onset type of Pompe disease may not become apparent until later in childhood, adolescence, or adulthood. Late-onset Pompe disease is usually milder than the infantile-onset forms of this disorder and is less likely to involve the heart. Most individuals with late-onset Pompe disease experience progressive muscle weakness, especially in the legs and the trunk, including the muscles that control breathing. As the disorder progresses, breathing problems can lead to respiratory failure.
Other names for this condition:

• 
  acid maltase deficiency
  acid maltase deficiency disease
  alpha-1,4-glucosidase deficiency
  AMD
  deficiency of alpha-glucosidase
  GAA deficiency
  glycogen storage disease type II
  glycogenosis Type II
  GSD II
  GSD2
  

I am no medical expert here hehe, but thought it could be helpful to look more into this disease. And all in all the movie was very good, the message I think was the important thing. When all the doctors the family had seen just told them to live the last moments with their child's to the fullest, they kept on going and fighting and gave their children more hope to have a better life :) The closing scene and song are just perfect and will make you teary eyed hehe:

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