Therapeutic Strategy
a Precise, Biology-Driven Approach
GG’s care integrates symptom management of today with molecular strategy for her future.
Clinically, her care is managed through coordinated efforts; neurology, immunology, gastroenterology, cardiology, nutrition, and metabolic oversight, including biologic therapy for immune-mediated inflammation and structured nutritional and IV metabolic support. These interventions have resulted in measurable improvements in gastrointestinal health, systemic inflammation, and functional capacity.
From a translational standpoint, the therapeutic objective is clear:
restore or enhance functional NAA15 expression from the intact allele.
Potential strategies under consideration include:
Antisense oligonucleotide (ASO)–based modulation - targeting increasing expression from the healthy copy of NAA15 that GG has intact
