Patient Stories

Individualized Treatment for Individual Patients

We can do this! The technology is already here. n-Lorem and its partners are ready to evaluate patients with rare genetic diseases who may benefit from treatment with custom antisense oligonucleotide (ASO) medicines.

Child with fatal brain disease treated with experimental ASOs

A child who experienced progressive loss of vision, epilepsy and neurological deterioration was found to have a unique mutation in a gene caused a variation of battens disease. The child has been treated with an experimental ASO and the child’s disease may be progressing less rapidly.

A child with progressive loss of vision and the ability to walk treated with an experimental ASO

A child born with an inability to feel pain experienced progressive deuteriation in vision and was found to have a unique mutation that causing posterior column ataxia and retinitis pigmentosa (PCARP) is receiving an experimental ASO treatment.