Extraordinary Measures
Name _________________________ Per _______
1. ________________________ What is the disease that the Crowley children suffer from?
2. ________________________ What kind of deterioration does the disease cause in all parts of the body?
3. ________________________ What are two common symptoms that people with this disease suffer from?
________________________
4. ________________________ This disease is considered what kind of a disorder?
5. ________________________ What are people suffering from this disease missing? (Processes sugar.)
6. What happens to the extra sugar in the bodies of people with this disease?
7. Why were enzyme injections unsuccessful in the treatment of this disease?
8. How was the enzyme Dr. Stonehill was working on different from those that had been created earlier?
9. The largest problem Dr. Stonehill encountered in doing his research was getting adequate ______________?
10. Why was it decided that the clinical trial for the drug created to treat Pompe Disease would only be given to
infants?
11. ________________________ Was Dr. Stonehill’s enzyme the only one chosen to participate in clinical trials?
12. What is a sibling trial?
13. Explain why Megan and Patrick began laughing after they were injected with the trial medicine?
14. What did this indicate?
15. At this time, the medicine for Pompe Disease can be administered to infants. How is it able to affect the
progression of the disease?
16. ________________________ After additional study laboratory research has shown that Dr. Stonehill’ theory
was correct. (True/False)