RUTLAND — The mother of two Saxtons River boys who have a rare terminal illness who launched an online campaign last year to gain access to a lifesaving drug is taking her fight to Congress.
Jennifer McNary is in Washington, D.C., today to present a petition with more than 170,000 signatures to the Food and Drug Administration, urging it expedite the approval of the drug eteplirsen. It has been called “a miracle drug” by families affected by Duchenne muscular dystrophy, a degenerative disorder in which a mutation causes the lost of dystrophin, a large-structure protein that protects muscle fibers.
“We are excited for the support we have received,” said McNary during a phone interview Tuesday.
She will be joined by several families also affected by DMD and by Rep. Michael Fitzpatrick, R-Pa., during a news conference today at the Rayburn House Office Building in hopes of moving the FDA to approve the drug.
Fitzpatrick said in a statement: “I have reached out to the FDA and urged them to consider placing these drugs into their Accelerated Approval Program. This program prioritizes the approval of the most effective drugs to provide greater relief to those diagnosed with serious illness like Duchenne muscular dystrophy. I remain hopeful that this expedited process will make these medical innovations accessible to the entire DMD community.”
McNary’s sons, Austin and Max, 14 and 11 respectively, were diagnosed more than a decade ago with Duchenne. Over the past year Max has had access to the drug eteplirsen as part of a highly successful clinical trial. The drug helps fight the loss of dystrophin by reconnecting the sequence of 79 exons by skipping over the one the boys are missing.
There is no cure for Duchenne, but since Max has been in the clinical trial, he has regained a lot of his strength and no longer needs to use a wheelchair, according to McNary. Austin was not allowed to participate in the trial.
“Max is not much of speaker and handshaker, so he and I walk to the zoo and walked back,” his mother said. “Max could not be better. He is continuing to amaze us.”
Austin, on the other hand, is getting weaker, but he has high hopes the drug will be approved soon, McNary said.
“Austin has become a great advocate,” she said of her older son. “He has come a long way... but he understands this is his life.”
McNary said Austin and his friend Jake Wesley, 12, who also has Duchenne, spent the beginning of the week visiting with members of Congress, including Sen. Patrick Leahy, D-Vt.
“They surprised me, knowing they are advocating for themselves,” she said.
McNary said the support they have received from people and organizations around the world has been amazing. She said she was always hopeful that people would jump on board and help them in any way they could.
“We want people to know how grateful we are,” she said. “This story is not over.”
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