Our commitment to rare and life-threatening NEUROLOGICAL genetic diseases

We work relentlessly to bring gene therapy to patients and families affected by rare and life-threatening neurological genetic diseases, like spinal muscular atrophy (SMA), Rett syndrome (RTT), and a genetic form of amyotrophic lateral sclerosis (ALS). Our dedication to advancing gene-based medicine can make a difference in the fight against devastating hereditary diseases.


When exploring different treatment options for your loved one, it's important to understand how gene therapy works. Gene therapy addresses the root cause of a genetic disease by replacing the function of a missing or faulty gene with a new working copy of the gene in order to restore the function of the protein.

The major steps of gene therapy are:

Visit www.exploregenetherapy.com for more information.