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MYR-101

It is caused by a mutation in the aspartoacylase gene (_ASPA_), which leads to a deficiency of the aspartoacylase enzyme (ASPA). … [L43567] The FDA has granted MYR-101 Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for the treatment of patients with Canavan disease.[L43572]

Irsenontrine

Irsenontrine is under investigation in clinical trial NCT03467152 (Study to Evaluate the Efficacy, Safety and Tolerability of E2027 (Hereinafter Referred to as Irsenontrine) in Participants With Dementia

AZD8630

AZD8630 is an experimental drug under investigation, as described in the Phase I clinical study titled "Randomised, Blinded, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics

Elezanumab

Elezanumab is under investigation in clinical trial NCT03737812 (A Study to Assess the Safety and Efficacy of Elezanumab When Added to Standard of Care in Progressive Forms of Multiple Sclerosis).

Afabicin

Afabicin is under investigation in clinical trial NCT03723551 (Study to Assess Safety, Tolerability and Efficacy of Afabicin in The Treatment of Participants With Bone or Joint Infection Due to Staphylococcus

BB 21217

BB 21217 is an anti-BCMA CAR T cell therapy that uses the same CAR molecule as [idecabtagene vicleucel] (bb2121), but adds the PI3K inhibitor bb007 during ex vivo culture to enrich the drug product (DP … ) for memory-like T cells.

Pipequaline

Pipequaline has a similar pharmacological profile to the reported for the benzodiazepines. … Due to these differences, this drug is classified as a nonbenzodiazepine anxiolytic.

AEG35156

XIAP interferes with both the intrinsic and extrinsic program-death signaling pathways, which may render tumor cells resistant to apoptosis. … This agent reduces total levels of XIAP in tumor cells, working synergistically with cytotoxic drugs to overcome tumor cell resistance to apoptosis.

Tyramine

An important characteristic of this product is its impediment to cross the blood-brain barrier which restrains its side effects to only nonpsychoactive peripheral sympathomimetic effects. … Tyramine acts by inducing the release of catecholamine.

Iprindole