151 president donald trumps first address to a joint session of congress happened to fall on rare disease day a fact he highlighted by recognizing one of his invited guests megan crowley a 20yearold college student with a rare genetic disorder called pompe disease after spotlighting crowley the president announced his intention to reduce restraints at the us food and drug administration fda in order to speed up the drug approval process for diseases like pompe if we slash the restraints not just at the fda but across our government then we will be blessed with far more miracles like megans he said heres a look at pompe disease and the drugs approved to treat it through the current fda drug approval process factchecking president donald trumps address to a joint session of congress what is pompe disease the disease is a rare genetic disorder where the body is unable to make an enzyme that breaks down a specific complex sugar called glycogen as a result an abnormal amount of the sugar accumulates especially affecting muscle cells the disease affects approximately 1 in every 40000 people in the us according to the national institutes of health nih there are three types of pompe disease classic infantileonset nonclassic infantileonset and lateonset classic infantileonset pompe disease begins within a few months of birth and is characterized by muscle weakness poor muscle tone enlarged liver and heart defects that if untreated will likely to lead to heart failure nonclassic infantileonset pompe disease usually appears by the time the infant is a year old and has a more subtle presentation it is characterized by delayed motor skills and progressive muscle weakness the heart may be abnormally large but this defect is less likely to lead to heart failure the third type of the disease called lateonset type of pompe disease may not show up until late childhood adolescence or adulthood it is generally milder characterized by progressive muscle weakness especially in the legs and the trunk including the muscles that control breathing butthe heart is much less likely to be affected megan crowley was diagnosed with pompe disease at 15 months her younger brother patrick was found to have the disease when he was just 3 months old how was the treatment developed during his speech trump used johny crowleys story as an example of how government restraints should be cut to facilitate innovation megans story is about the unbounded power of a fathers love for a daughter trump said but our slow and burdensome approval process at the food and drug administration keeps too many advances like the one that saved megans life from reaching those in need at the time the crowley children were diagnosed with the disease there were few options for treatment john crowley decided to quit his job at bristolmyers squibb in order to search for a cure and cofounded novazyme pharmaceuticals in oklahoma city with dr william canfield who was researching pompe disease in 1999 the