Tonus Therapeutics

Tonus Therapeutics
8210 Golden Oak Circle
Williamsville NY 14221


Muscular dystrophy is a defect in cell mechanics. It is caused a genetic defect in the DNA coding for the protein dystrophin. Dystrophin forms a network of protein strands that reinforce the cell membrane. With the loss of support, stress in the membrane

sampleJB on his first day of school.
increases, causing mechanosensitive ion channels to open, letting calcium leak into the muscle. This appears to cause the muscle degeneration associated with muscular dystrophy.

There are many mutations in DNA that can produce muscular dystrophy and researchers are working on methods to repair the DNA. However, reliable DNA treatments are still in the future, changing DNA can lead to immune reactions, and children need therapies now.

We have found a small protein (about half as big as insulin) that can inhibit the calcium influx associated with muscular dystrophy. This offers a possible therapy that does not rely on addressing any specific DNA mutation.

A treatment, not a cure. It is important to understand that our drug, called GsMTx4, is a promising but not yet proven therapy to improve the mobility of boys with dystrophy. We hope that by slowing the rate of degeneration, natural growth process will cause the boy's mobility to improve, plateau. or at least slow the rate of atrophy.

Patented, approved by the FDA as a orphan drug.
Orphan drugs, those that apply to a relatively small number of patients, may be approved more rapidly by the FDA for clinical trials.

We are currently raising funds for the pre-clinical testing required by the FDA and we will then seek IND (investigative new drug) approval to allow it to enter clinical trials. We hope to begin bring GsMTx4 to clinical trials within 2 years. We will announce on this website when clinical trials begin and we are recruiting candidates for the trials.