While the gene therapy could enhance cognitive abilities and prolong skills for Emmett, it would not reverse existing damage. His family stresses the importance of maintaining his current skills and making the most of his childhood.

The costs for such gene therapies are expected to be very high, often exceeding $3 million, posing significant financial barriers for families seeking access.

The prognosis for Emmett was initially grim, with his family believing he might die young, but the potential availability of gene therapy has shifted their outlook, motivating them to focus on making the most of his childhood.

Early intervention is critical, but the delay in approval raises concerns about children losing essential skills before treatment can be administered.

Research for Sanfilippo treatments remains in early stages globally, mainly in pre-clinical phases, with no current cure available. In Canada, efforts are focused on preventing hearing loss, and treatments are still in early development.

A family in Saskatoon is hopeful about a new gene therapy for their six-year-old son Emmett, who has Sanfilippo syndrome, a rare and terminal genetic disorder often called 'childhood Alzheimer's.' They are actively advocating for access to this U.S.-approved treatment, which could significantly improve his quality of life, although it has not yet been submitted to Health Canada.

The FDA has granted accelerated approval for the gene therapy, but the process has been delayed until 2026 due to manufacturing concerns, leaving the family hopeful but awaiting access.

Getting approval and funding for the therapy in Canada involves navigating government processes, with the Saskatchewan Ministry of Health indicating coverage is possible but requires prior approval and funding arrangements.

Sanfilippo syndrome causes delayed development, regression, and loss of speech and mobility, with most affected children living only into their mid to late teens. Currently, there is no cure.

The family highlights that access to this potential treatment has changed their outlook, shifting their focus from grief to making the most of Emmett's childhood despite the prognosis.