申请人:ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
公开号:US20220226289A1
公开(公告)日:2022-07-21
Methods and formulations are provided for treating spinobulbar muscular atrophy in a subject in need thereof. By administering a therapeutically effective amount of a selective androgen receptor modulator or a small molecule such as 1-[2-(4-methylphenoxy)ethyl]-2-[(2-phenoxyethyl)sulfanyl]-1H-benzimidazole or a derivative, prodrug, or pharmaceutically acceptable salt thereof, one or more symptoms of spinobulbar muscular atrophy can be ameliorated. The effective amount can be effective to prevent or delay loss of body weight, a loss of mobility, and/or a loss of physical strength in the subject; to prevent or delay neurogenic atrophy and/or to prevent a loss of spinal cord motor neurons in the subject; to restore the frequency of type I myofibers to normal levels for a healthy subject; and/or to reverse testicular atrophy in the subject.