Neurofibromatosis (NF) is a genetic disorder that leads to the development of tumors on various nerve tissues. These are usually benign, but it can turn malignant in some cases.
Friedreich’s Ataxia (FRDA) is an inherited disease of the central nervous system. It is the most common autosomal recessive ataxia caused by an abnormality of a single gene called the Frataxin FXN gene.
Post-traumatic stress disorder (PTSD) is a neurological disorder that develops in people who have experienced a shocking, scary, or dangerous event. It usually occurs more often in women than in men.
Post-operative pain is a very common problem after any surgery and it is necessary to treat the same effectively, so that the patient feels more comfortable. According to the American Society of Anesthesiologist practice guidelines for acute pain management in the perioperative setting, acute pain is defined as pain that is present in a surgical patient after a procedure.
Progressive supranuclear palsy (PSP), also known as Steele-Richardson-Olszewski syndrome, is a rare brain disorder, which affects behavior and thinking capability of an individual.
NF-κB is identified as a family of highly conserved transcription factors, which are responsible for many cellular functions such as regulation of inflammatory responses, apoptosis, cellular growth, and others.
IL-6 is transcribed by IL-6 gene that encodes a cytokine, which functions in inflammation and maturation of B cells. The IL-6 protein can induce fever in patients suffering from autoimmune diseases, thereby acting as endogenous pyrogens.
Restless legs syndrome (RLS) can be described as a neurological sleep disorder causing intense and irresistible urge in moving legs. It can either occur genetically or as a result of various medical problems such as Parkinson’s disease, iron deficiency, diabetes, peripheral neuropathy, and others. Symptoms of this disease include itching, aching, crawling, burning, throbbing, tingling, and creeping.
LGS is a severe form of epilepsy that occurs rarely and mostly affects pediatrics. This disease has the tendency to develop between the age of one to eight years in children, and can be characterized by polymorphic seizures and neuropsychological decline. The exact cause of this syndrome is unknown. It may be symptomatic, which is secondary to an underlying brain disorder.
Mitochondria are the organelles found in cytoplasm of most eukaryotic cells. They are also known as power-house of the cell. Various mitochondrial-related diseases have been discovered, in which the pathogenesis is not understood well.
Galectins belong to the family of animal lectins and perform function by interacting with cytoplasmic and nuclear proteins, cell-surface and extracellular matrix glycoproteins and glycolipids to modulate signalling pathways.
Epidermal growth factor receptors (EGFR) are a group of receptor tyrosine kinase (RTK) that plays a major role in cell proliferation, survival and differentiation. In most of the cancer, EGFR are amplified and overexpressed, due to dysregulation, resulting in cancer development.
Mitogen-activated protein kinase (MAPK) are ubiquitous regulators of many cellular functions including cell proliferation, cellular growth, inflammatory responses to stress signals and cell differentiation.
MDM2 protein are powerful oncogene which is overexpressed in various cancers, including breast cancer and sarcoma. There are many small molecule drug candidates that are being developed as MDM2 protein inhibitors as monotherapy or combination therapy for the treatment of various cancers.
Interleukin receptors are a group of cytokine receptors that are expressed by leukocytes. Interleukin receptors play an important role in the functioning of the immune system.
Interleukin 8 is a chemokine produced by various immune cells. They promote angiogenesis, proliferation and development of cancerous cells. The other diseases which are associated with interleukin 8 are depression, schizophrenia, bipolar disorder, migraine, Alzheimer’s disease, colitis, rheumatoid arthritis, multiple sclerosis, asthma, allergic rhinitis, psoriasis, eczema and others.
Hepatocyte growth factor (HGF) is secreted by human pancreatic stellate cells and present predominantly on cancer cells. Thus, HGF inhibitors has emerged as an effective therapy in preventing local tumor growth.
Glycogen synthase kinase 3 beta (GSK-3β) is an attractive target for the treatment of psychiatric disorders and neurodegenerative diseases. Many GSK-3β inhibitors have been developed for the treatment of different central nervous system disorders.
Tau protein is found in abundance in neurons of the central nervous system. This protein is a product of microtubule-associated protein tau (MAPT) gene, that is located on chromosome 17. It is associated with pathologies of several nervous system disorders, such as dementia, Parkinson’s disease, and Alzheimer’s disease.
RNA interference (RNAi) is a molecule type that silences the gene and limits the transcription of the mutated gene. Gene silencing is a novel mechanism that inactivates the transcripts of mutated gene, by activating sequence specific RNA degradation process.
Rho kinase inhibitor, also known as ROCK inhibitor, inhibits the rho kinases, a family of small GTP-binding protein. Rho kinases are found to regulate cell motility, proliferation, shape, gene expression, apoptosis, and are also involved in the signalling pathway.
Nav1.7 voltage-gated sodium channel inhibitors are the drug candidates that target the sodium channels Nav1.7 and have been significant in pain management. The sodium channels Nav1.7 receptors are generally found in two types of neurons: nociceptive pain neurons, such as trigeminal neurons and dorsal root ganglion; and sympathetic ganglion neurons which form a part of the autonomic (involuntary) nervous system. These receptors play an important role in generation and conduction of action potential.
Cyclophilin befits in the group of protein that shows peptidyl-prolyl cis-trans isomerase activity, found in all types of cells. In humans, 16 cyclophilins have been identified till date. Cyclophilin A, a member of cyclophilin group, mediate the action of immunosuppressive drugs.
Cell cycle inhibitors include cyclin inhibitors and cyclin-dependent kinases (CDKs), which plays major role in developing new class of anti-cancer therapies. Also, cell cycle inhibitors in combination with chemotherapy, overcome drug resistance and improve cytotoxic efficacy. CDKs are rational targets for cancer treatment, that could restore cell-cycle checkpoints and may induce apoptosis.
Brain-derived neurotrophic factor (BDNF) is a type of nerve growth factor that stimulates the survival of dorsal root ganglions. The BDNF gene comprises of five exons, including four exons with distinct promoter and fifth exon transcript with the mature BDNF protein.
Aurora kinase are proteins that function as mitotic regulators and are aberrantly expressed in cancerous cells. Aurora kinase inhibitors target these aberrantly expressed regulators, provides genetic stability and prevents tumorigenesis.
Adenosine 5-monophosphate activated protein kinase (AMPK) plays an important role in regulation of homeostasis. Dysregulation of AMPK can result in many diseases which includes obesity and type 2 diabetes. Several studies have also suggested that AMPK are also associated with the development of neurological diseases and cancer.
Androgens are responsible for the development of male characteristics by binding to androgen receptors. Androgen receptor plays an important role in the development and progression of prostate cancer. Thus, it has been an attractive target for the clinical interventions and treatment of prostate cancer.
Alpha-synuclein is a protein that is abundantly found in the brain. The minimal amount of alpha-synuclein is also found in the heart and other tissues. The protein is prominently found at the tips of the nerve cells. It helps in the maintaining supply of the synaptic vesicle in presynaptic terminals. In addition, the alpha-synuclein protein also helps in release of dopamine.
Tosymra, previously known as DFN-02, an intranasal spray containing sumatriptan (10 mg) and a 5-HT1B/1D receptor agonist developed by Dr. Reddy’s Laboratories Limited and its subsidiary Promius Pharma, LLC, is approved by the USFDA in January, 2019 for the acute migraine.
Sufentanil, sold under the brand names of Dsuvia and Sufenta, is basically a synthetic opioid analgesic drug, which is used for the medication of acute pain relief. It helps to offer properties of sedation and this makes it a good analgesic component of anesthetic regimen during a surgical process.
Lumateperone by Intra-Cellular Therapies Inc., has multiple mechanisms, such as that of a potent serotonin 5-HT2A receptor antagonist; a dopamine receptor phosphoprotein modulator (DPPM), acting as a presynaptic partial agonist and postsynaptic antagonist at dopamine D2 receptors, a dopamine D1 receptor-dependent indirect modulator of glutamate (both NDMA and AMPA), and a serotonin reuptake inhibitor.
Akt, also known as PKB, is generally activated in several types of cancers such as breast, gastric, lung, ovary, and pancreatic cancer. Akt inhibitor prevent the functioning of activated form of Akt kinases. Akt kinases are also involved in promoting growth factor-mediated cells that mediates cell proliferation and inhibition of apoptosis through the inactivation of pro-apoptotic proteins, such as mouse double minute 2 homolog (MDM2) and Bcl-2 antagonist of cell death.
Visceral pain is the most frequent form of clinically relevant pain for those patients seek medical attention. There are several causes of visceral pain. Nociceptive pain causes direct injury of an internal organs, that leads to cardiac ischemic, and peptic ulcer. Medications for visceral pain treatment includes analgesics such as nonsteroidal anti-inflammatory drugs, paracetamol and serotonergic compounds.
Tay-Sachs is a lysosomal storage disorder caused by the deficiency of vital enzymes called beta-hexosaminidase A (Hex-A). The role of Hex-A is to degrade a fatty substance or lipid called GM-2 ganglioside.
Tardive dyskinesia (TD) is an involuntary neurological movement disorder caused by the use of dopamine receptor blocking drugs which are prescribed to treat certain psychiatric or gastrointestinal conditions. Long-term use of these drugs may produce biochemical abnormalities in the area of the brain known as the striatum.
Major depressive disorder, also known as the clinical depression, is characterized by a constant sense of despair and hopelessness. It usually affects daily activities such as study, sleep, and eating.
Ischemic stroke develops due to an obstruction within a blood vessel which supply blood to the brain. The underlying condition for this type of obstruction is atherosclerosis, which is characterized by fatty deposition or plaque build-up in the blood vessels.
Infantile spasm, also known as West syndrome, is a rare type of seizure disorder which is characterized by sudden stiffening of the body, legs and arms, and forward bending of head. It is diagnosed by physical examination, neurological examination, electroencephalography (EEG), magnetic resonance imaging (MRI), blood tests and urine tests.
Hypersomnia is a medical condition which is characterized by excessive daytime sleepiness. It is categorized into primary hypersomnia and secondary hypersomnia. People with hypersomnia also suffer from other sleep-related problems, such as clear thinking problems and lack of energy.
Hyperalgesia is a medical condition in which a person develops an increased sensitivity towards pain. There are several nerve pathways in the body where signals can start miscommunication with each other, resulting in hyperalgesia.
Essential tremor, a nerve disorder, is characterized by involuntary tremors or shaking in different parts of the body. The most common affected areas are head, arms, hands, larynx, chin and tongue. This disease creates difficulties in daily activities such as eating, writing and dressing.
Dravet syndrome is a form of severe myoclonic epilepsy that occurs in infants during their first year of age. If left untreated, it can be associated with other types of seizures, and status epilepticus. Children having Dravet syndrome are generally observed with poor cognitive skills and hyperactivity.
Dementia is a group of neurological disorders which is characterized by impaired intellectual functioning. People having dementia generally lose the ability to maintain emotional control and solve the problems.
Complex regional pain syndrome (CRPS) is a type of chronic pain that affects one limb of the body (hand, leg, arm, or foot). The condition generally arises due to severe injuries or malfunctioning of central and peripheral nervous systems. CRPS is categorized into two main types: CRPS-I and CRPS-II.
Cognitive impairment is a medical condition in which people lose their cognitive capabilities such as thinking skills and memory. People having cognitive impairment are at high risk of developing dementia or Alzheimer’s disease. Cognitive impairment that affects memory is known as amnestic mild cognitive impairment (MCI); and the condition which affects thinking skills called nonamnestic MCI.
Chronic and low back pain is a constant and dull ache and creates a sharp sensation that leaves a person harmed. It can occur due to sciatica, spondylolisthesis, spinal stenosis, traumatic injury, herniated or ruptured discs, and intervertebral disc degeneration.
In cerebral palsy, the term cerebral refers to brain and palsy refers to the loss or impairment of motor function. Cerebral palsy is a group of neurological disorders that appear in infancy or early childhood, and permanently affect body movement and muscle coordination.
Asperger syndrome is an autism spectrum disorder characterized by impaired communication skills and repetitive thoughts or behavior. Symptoms associated with the Asperger syndrome are lack of speech recognition skills, dislike for routine changes, avoidance of eye contacts, unusual facial expressions, and delayed motor development.