To provide the FDA with a comprehensive understanding of Myhre syndrome, including the lived experiences of patients and caregivers, symptom burden, disease progression, and the community’s perspectives on treatment priorities and clinical trial participation, families, clinicians and patients met with the FDA in December 2024. The published report can be found here.

Zac Burns, a member of the Myhre Syndrome Foundation, summarized that Myhre Syndrome is a systemic disorder with heterogeneous symptoms among the patient population. As such, it’s impossible to represent the interests of all the patients or discuss the burden of every symptom. However, the seven patient and caregiver testimonials reveal common themes and Myhre syndrome features that are the highest priorities for treatment.