Yadilette's research focuses on treating lysosomal storage disorders, which cause bone abnormalities, through enzyme replacement therapy rather than bone marrow transplantation. Her treatment involves purifying proteins through crystallization and x-ray analysis to determine the replacement enzyme, then injecting patients weekly and monitoring their progress through tests, with some patients showing increased mobility based on walking and climbing tests. This research helps improve the lives of those suffering from these diseases and creates more awareness.

1. Reflection on the third seminar:
Yadilette’s work concentrates on lysosomal storage disorders, which cause
substrate accumulation and lead to improper development of bones. As said by the
article, these disorders are majorly treated with bone-marrow transplantation. However,
her research treatment for this is the use of proteins as drugs. To determine the enzyme
replacement, crystallization is done. This consists of purifying the protein, passing
through processes that form a crystal, freezing it with liquid nitrogen, directing x-rays at
it and creating a map of the protein using the dots that are reflected off the crystal. The
treatment is injected in the patient once a week and then some tests are run to observe the
progress. “Patients who were randomized to galsufase and who continued to receive it
showed increases in the distance walked in 12 minutes and in the rate of stair climbing
compared with the start of the open-label period.” John J. Hopwood, et al. 2006. This
research helps the life of those who suffer from these diseases and it helps create
awareness of it.