Background: In the U.S., about 53 million informal, unpaid caregivers provide care to a person who is ill, is disabled, or has age-related loss of function. T…

Inherited Metabolic DisordersUndiagnosed DiseasesBatten's Disease+2 more

~Estimated

~$10 – $675

National Human Genome Research Institute (NHGRI)NCT05007990

Phase 1

Phase 1/2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9

The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neurona…

CLN6Batten DiseaseBatten's Disease+2 more

~Estimated

~$10 – $6,250

The Charlotte and Gwenyth Gray FoundationNCT07582484