NRC-AN-019 has been found to be a promising new lead compound for the therapy of imatinib mesylate-resistant chronic myeloid leukemia. NRC-AN-019 showed considerable safety and response. In addition, it has the therapeutic potential in the treatment of Her-2-positive breast cancer.

Florilglutamic acid enters cells through a transport system known as xC- (cystine/glutamate antiporter) which is more abundant in cancer tissue. Florilglutamic acid (18F) is a radioactive compound for use with an imaging method known as positron emission tomography (PET). When injected into the patient, florilglutamic acid (18F) is more effectively taken up by the cancer cells in the liver from where it is expected to emit radiation that can be detected in a PET scan, thereby allowing the cancer to be diagnosed. Orphan designation (EU/3/16/1632) was granted by the European Commission to Piramal Imaging GmbH, Germany, for florilglutamic acid (18F) for the diagnosis of hepatocellular carcinoma.

GUANOSINE 5'-DIPHOSPHO-β-L-FUCOSE is classified as a member of the Purine nucleotide sugars. Guanosine 5'-diphospho-β-L-fucose sodium salt sodium salt is a substrate for fucosyltransferase.

MCD-386CR (CDD-0102A) is a functionally selective agonist with partial agonist activity at M1 muscarinic receptors, weak activity at M3 receptors, and no activity at other muscarinic receptor subtypes. In preclinical studies, it enhances memory function in animal models of Alzheimer's disease. MCD-386CR (CDD-0102A) received orphan drug designation from FDA in 2011. The drug is intended to treat progressive supranuclear palsy, a degenerative brain disease that affects balance, walking, speech, cognition, and eye movements.

Carbovir is a nucleoside reverse transcriptase inhibitor analog of guanosine. Carbovir decreases HIV viral loads, retards or prevents the damage to the immune system, and reduces the risk of developing AIDS. Carbovir Triphosphate belongs to the class of organic compounds known as nucleoside and nucleotide analogues. These are analogues of nucleosides and nucleotides. Carbovir interferes with the enzyme HIV uses to manufacture new viral particles within an infected cell, and is primarily metabolized to the 5'-triphosphate of Carbovir (CBV-TP) to concentrations sufficient to inhibit HIV reverse transcriptase.

RG3039, also known as PF-06687859, is the first small molecule developed specifically for treatment of Spinal muscular atrophy (SMA). This drug is used in SMA patients that have reached early stage clinical trials. It was shown that RG3039 improved survival, function and motor unit pathologies in SMA mouse models. It is known, that RG3039 is a potent inhibitor of the mRNA decapping scavenger enzyme (DcpS). DcpS is a nuclear shuttling protein that binds and hydrolyzes the m(7)GpppN mRNA cap structure and a modulator of RNA metabolism. Therefore, DcpS represents an intrigueing therapeutic target for modulating gene expression by a small molecule. The exact therapeutic mechanism remains unknown.