New Hope for Sheridan Family
by Nancy Banister
June 5, 2003 issue of The Madisonian
On April 30, 2003, the FDA approved the use of Enzyme Replacement
Therapy (ERT) for victims of a genetic disease called
mucopolysaccharidosis (MPS). With her husband, Dan, needing to
remain in Montana, Linda Day and her three children, Danette, Scott
and Greg visited Washington DC in January to testify before an FDA
advisory committee asking them to recommend approval of the biologic
Both Scott and Greg have MPS 1-Hurler-Scheie syndrome, a genetic
disorder which causes a lack of an enzyme in the body which breaks
down sugars, eventually accumulating in the body. Five years ago,
Greg's health was plummeting. Frequent hospitalizations and heart
surgeries were contributing to his premature death. Then hope came
when Greg was accepted into a clinical study started by Dr. Emil
Kakkis at UCLA in Los Angeles. Because only one sibling per family
would be accepted in this study, Scott chose not to apply. He was
afraid his brother might not live long enough for FDA approval for
Greg has responded well after five years of treatment. He was
able to finish college Summa Cum Laude and live a better life.
Unfortunately after five years, the treatment was still unavailable
for Scott because the FDA did not approve it. Now Scott's health is deteriorating.
When his mother appealed to the FDA committee for approval of ERT,
she knew that this was the best and only treatment available.
Part of Linda's speech to the committee reveals the family's
concern for Scott. "It feels like we are walking on eggshells
waiting for the 'process' of approval. It is ever closer but still
out of reach. Scott's life is completely dependant on you (the
FDA committee) and your recommendation. Life and the quality of
life are being lost everyday that this treatment is not
available." The advisory committee voted unanimously to
recommend the FDA approval of ERT.
Dan and Linda are overjoyed that Scott can now start treatments
to stop the progress of his disease. He will be receiving treatments
from Dr. Ron Loge at Barrett hospital in Dillon. They would like to
share their joy with the great community who has supported them for
a quarter of a century. They will be having a potluck picnic at
their house on Indian Creek Road on June 15 at 4pm. Please bring
lawn chairs and come celebrate life with them.
© Copyright Nancy Banister and The Madisonian, reproduced by permission.