Families and clinicians press lawmakers on Prader-Willi care, waivers and treatments

Dr. Michael Gambello (Emory University) told the committee Prader-Willi syndrome (PWS) is a rare genetic disorder that disrupts hypothalamic function, causing infancy hypotonia and, later, an insatiable appetite (hyperphagia) that can lead to severe obesity and medical complications. He emphasized the need for coordinated specialty care across endocrinology, behavioral health, nutrition and social services and said consistent access to specialists and early diagnosis improves outcomes.

Family members and state advocates described day-to-day caregiving challenges, obstacles obtaining and retaining Medicaid and waiver services (including Katie Beckett), and clerical errors that can terminate coverage. Lisa Mataszak, president of the Georgia Association for Prader Willi Syndrome, and other parents urged policy solutions such as better waiver capacity, streamlined appeals, and consideration of automatic waiver qualification for PWS.

Karen Knight Hinkley (Solano Therapeutics) told legislators that her company recently secured FDA approval for the first drug to treat hyperphagia in PWS for ages 4 and up and said Solano committed $5,000,000 to research grants in the area. Family advocates asked the committee to prioritize targeted, actionable requests (for example, waiver access and expedited appeals) so the legislature can pursue feasible, near-term fixes.

The chair invited families to supply contact information and offered office help to investigate individual Medicaid terminations; committee members encouraged ongoing engagement and suggested follow-up briefings focused on a limited set of policy asks.