Charley’s Fund has collaborated with Galapagos, a Belgian biotech company, to develop the company’s SARM drug (selective androgen receptor modulator) as a Duchenne treatment. Galapagos was already developing the drug for cachexia (cancer-induced muscle wasting). Charley’s Fund supported an efficacy study and a dose ranging study in the mdx mouse to determine if this SARM might benefit boys with Duchenne, who also experience muscle wasting. Positive results in both studies
have spurred us to continue the collaboration and start planning for a clinical trial in the Duchenne population.