The Characters
This scientist is known for his unconventional methods and plays a key role in developing a treatment.
Dr. Robert Stonehill
This government agency must approve new treatments before public use in the U.S.
FDA
This is the main reason John Crowley starts a biotech company.
to save his children
Megan's favorite animal?
Penguin
List one major challenge involved in getting Stonehill's enzyme to clinical trials.
money, time, competition, regulations
These two children suffer from Pompe disease.
Meghan & Patrick
Which chromosome carries the affected gene causing Pompe?
Chromosome 17
Why are rare diseases harder to study and discover treatments for?
fewer patients= limited funds
What color does Megan want the medicine to be?
Pink
What ethical issue(s) arise when deciding who gets access to experimental treatments?
Infants are a better choice; conflict of interest with John as CEO
This trait best describes John Crowley’s actions throughout the film.
determination/resiliance
never giving up
This body system is primarily weakened by Pompe disease.
Muscular System & Cardiovascular System
According to Pete (John's coworker), how many biotech companies fail out of 10?
9/10
This specific enzyme is missing or not working properly in Pompe disease.
GAA
What is Dr. Stonehill's favorite hobbie/free-time?
Bass fishing
Which character represents the corporate and financial interests behind the drug development process and eventually fires John?
Kent Weber
This part of the cell is affected in Pompe disease due to enzyme deficiency.
Lyosomes
the first step before testing a drug in humans?
lab testing or animal testing
This is the chance two carrier parents have of having an affected child.
25%
What university does Dr. Stonehill work for?
University of Nebraska
This actor portrays the eccentric scientist Dr. Stonehill?
Harrison Ford
This type of therapy is developed to treat the disease by replacing the missing enzyme.
Enzyme Replacement Therapy
At first the drug is only approved for infants—what is the loophole Stonehill suggests as a "Hail Mary"?
sibling trial
The field of science studies that focuses on diseases like Pompe.
Genetics
this molecule builds up in cells in Pompe disease due to an enzyme deficiency
glycogen