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Lovotibeglogene autotemcel

[L49266] It was approved alongside [exagamglogene autotemcel] (Casgevy), another gene therapy, with the two treatments comprising the first cell-based gene therapies for the treatment of SCD in patients … [A262736,L49256] On December 8, 2023, lovotibeglogene autotemcel was approved by the FDA under the brand name Lyfgenia for the treatment of sickle cell disease (SCD) in patients ages 12 and older who

ANA971

ANA971, an orally administered prodrug of isatoribine. Isatoribine is a nucleoside analog in development for the treatment of chronic hepatitis C virus (HCV) infection. … ANA971 is a prodrug designed to improve the oral bioavailability of isatoribine.

Arbaclofen

It is believed to be a selective gamma-amino butyric acid type B receptor agonist, and has been investigated as a treatment for autism spectrum disorder and fragile X syndrome in randomized, double blind … It has also been investigated as a treatment for spasticity due to multiple sclerosis and spinal cord injury.

ISIS 113715

In early trials, ISIS 113715 induced statistically significant improvements in multiple measures of glucose control along with statistically significant reductions in LDL-cholesterol. … ISIS 113715 is our second-generation antisense inhibitor of protein tyrosine phosphatase 1b, or PTP‑1b, for the treatment of type 2 diabetes.

FX06

FX06 is a naturally occurring peptide derived from the neo-N-terminus of fibrin (Bbeta(15-42)). It prevents leukocyte migration through the gap junctions of endothelial cells. … FX06 has proven safe in acute and subchronic toxicological studies and recently entered clinical development. It is being developed by Fibrex Medical Inc.

PYM50018

PYM50018 is a patented, orally active, neuroprotective and neuroregenerative compound. It is developed for the treatment of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease). … In pre-clinical models, PYM50018 has been observed to protect against neuronal damage, increase neurite outgrowth, reverse oxidative damage and reverse neuronal apoptosis in vitro.

Cefacetrile

A derivative of 7-aminocephalosporanic acid.

Cycrimine

Cycrimine is a drug used to reduce levels of acetylcholine to return a balance with dopamine in the treatment and management of Parkinson's disease.

Dansylamide

Stamulumab

MYO-029 is a human anti-GDF-8 monoclonal antibody, which is being developed to treat muscle-wasting diseases including muscular dystrophy and age-related sarcopenia.