For trehalose
Looks like its been used for numerous other disorders:
PHASE STATUS PURPOSE CONDITIONS COUNT START DATE WHY STOPPED 4 Completed Treatment Corneal De-epithelialization/Photorefractive Keratectomy 1 somestatus stop reason 4 Not Yet Recruiting Prevention Cataracts/Dry Eye Syndrome (DES)/Dry Eyes 1 somestatus stop reason 4 Not Yet Recruiting Treatment Parkinson's Disease (PD) 1 somestatus stop reason 3 Completed Treatment Bipolar Disorder (BD)/Depression, Bipolar 1 somestatus stop reason 2 Completed Treatment Muscular Dystrophy, Oculopharyngeal (OPMD)
Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood-brain barrier, stabilizes proteins and, importantly, activates autophagy, which is the process that clears pathologic material from cells. In animal models of ALS, treatment with trehalose delayed onset of disease, prolonged survival and preserved motor function and motor neurons in the spinal cord. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a myriad of diseases with pathologic accumulation of storage material. The drug is manufactured by Seelos Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases
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Ann: Monepantel Selected for Entry into HEALEY ALS Platform Trial, page-53
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