Ontario girl with Rett Syndrome denied access to promising gene therapy trial, parents say
Lucia Vaccaro, a four-year-old girl with Rett Syndrome, was initially selected for a promising gene therapy trial but later became ineligible due to changes in the trial's age requirements. Her parents attempted to access the therapy through Health Canada's special access program, but their request was denied without a clear explanation. The family is concerned about the lack of transparency and accountability in accessing emerging therapies for Canadians with rare conditions like Lucia's.
- ▪Lucia was diagnosed with Rett Syndrome, a neurodevelopmental disorder that affects motor skills and language.
- ▪The gene therapy trial initially included children aged two to six but later restricted eligibility to only two- and three-year-olds.
- ▪The Vaccaro family was denied access to the therapy through Health Canada's special access program, leaving them without treatment options.
Opening excerpt (first ~120 words) tap to expand
Open this photo in gallery:Rana and Joseph Vaccaro with their daughter Lucia, Ottawa. May 21.Dave Chan/The Globe and MailShareSave for laterPlease log in to bookmark this story.Log InCreate Free AccountRana and Joseph Vaccaro could barely contain their excitement inside a Montreal children’s hospital last December. Holding on to her four-year-old daughter, Ms. Vaccaro whispered “Finally, you’re in.”Their daughter, Lucia, was diagnosed two years earlier with Rett Syndrome, a rare and complex neurodevelopmental disorder that affects how a brain develops and progressively erodes motor skills and language.
…
Excerpt limited to ~120 words for fair-use compliance. The full article is at The Globe and Mail.
Discussion
0 commentsMore from The Globe and Mail
