Nonprofit foundation
Appears in 1 story · Last updated Sep 21, 2026
As of September 2026: Developed and supplied nL-CHCHD-001; now treating about 10 people
A physician with a rare inherited form of amyotrophic lateral sclerosis (ALS) received a drug built around his own gene mutation, the first therapy designed for this genetic form of the disease. A year after his first dose, his neuron-damage biomarker fell into the normal range and he kept practicing medicine.
Updated Sep 21
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