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DTx-1252

DTx-1252 is a siRNA therapeutic under investigation for the treatment of Charcot-Marie-Tooth Disease type 1A (CMTD1A).[L48096]

CSIC002

CSIC002 is a small-molecule Sema-3A inhibitor. It is being investigated for the treatment of anterior ischemic optic neuropathy.

Deanol

Deanol is commonly referred to as 2-(dimethylamino)ethanol, dimethylaminoethanol (DMAE) or dimethylethanolamine (DMEA). It holds tertiary amine and primary alcohol groups as functional groups.

Astodrimer

It is a lysine-based dendrimer with naphthalene disulfonic acid surface groups.

Olipudase alfa

[L42740] ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin … [A251590] It is the first and only enzyme replacement therapy in the world for the treatment of Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann–Pick disease.

AZX-100

TG-C

Fenretinide

A synthetic retinoid that is used orally as a chemopreventive against prostate cancer and in women at risk of developing contralateral breast cancer. It is also effective as an antineoplastic agent.

Benzoylecgonine

It is the main pharmaceutical ingredient in the investigational drug Esterom, a topical solution used for the relief of muscle pain that is not FDA approved or on the market in the United States.

V24343

The anti-obesity drug, V24343, acts by targeting the CB1 receptor in the brain and suppressing a person's appetite.