Phrixus Pharmaceuticals has announced that the first person living with Duchenne has completed 15 months of treatment with Carmeseal-MD. The treatment was well-tolerated; benefits in these patients included reductions in muscle damage markers such as creatine kinase and cardiac troponin.
“The strong preclinical data, combined with human safety data from over 2,500 patients in un-related indications creates a strong rationale for using P-188 NF, a membrane stabilizer, in DMD, a disease that is characterized by membrane tears due to the absence of functional dystrophin,” said Bruce Markham, Ph.D., Chief Scientific Officer and VP Research of Phrixus.
“Providing Carmeseal-MD as an unlicensed special provides an opportunity for specialty physicians outside of the United States to treat patients especially with regard to heart failure and respiratory dysfunction, the two leading causes of death,” added Thomas A. Collet, President & CEO.
Carmeseal-MD is available outside of the United States through Phrixus’s Expanded Access Program and Ethicor Pharma Ltd., a distributor.
About Carmeseal-MD
In animal models of Duchenne, Carmeseal-MD (Poloxamer 188 NF) has been shown to improve the efficiency of damaged hearts and the performance of damaged diaphragms with once-a-day subcutaneous administration at low doses.
When infused into the bloodstream, it encounters and binds to microscopic tears in the muscle and prevents the pathological leakage of calcium into the cells, which keeps the muscle from performing as required. Carmeseal-MD is expected to have its effect in patients with Duchenne irrespective of the genetic defect that causes the disease.
For more information contact info@actionduchenne.org
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