In A First, Orphan Drug Designation Granted for Treatment of Alagille Syndrome

Food and Drug Administration (FDA) has granted Orphan Drug designation to A4250 for the treatment of Alagille syndrome, a life-threatening and rare disease that primarily targets the liver. This genetic disorder affects approximately 1 in 70,000 newborns and is characterized by liver damage due to bile stasis (cholestasis) with signs and symptoms including jaundice, poor weight gain, loss of appetite, poor growth, and pruritus. Alagille (AGS) syndrome is variably characterized by cholestasis, peripheral pulmonary artery stenosis, characteristic facies, anterior segment abnormalities, pigmentary retinopathy, and dysplastic kidneys.

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Multiple Sclerosis and Sleep Disorders

Multiple sclerosis is an autoimmune inflammatory disease of the central nervous system. Around 400,000 people in the US are diagnosed with MS [1]; it is commonly diagnosed in early adulthood and is twice as common in women as men [2]. In MS, inflammation damages the protective coat around nerves (the myelin sheath), which reduces the efficiency of message transmission down those nerves.

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NEW GENETIC DISORDER REPORTED SIMILAR TO JANSEN METAPHYSEAL CHONDRODYSPLASIA

An intricate case of a family with 2 children with a rare skeletal disorder has been reported in October 2018. The disorder has not been reported before, and clinically, it appears similar to Jansen metaphyseal chondrodysplasia (JMC). JMC is an exceedingly rare disorder causing abnormal development of portions of the arm and leg bones. Unusual cartilage formations and subsequent abnormal formation of the long bones results in short arms and legs. Short-limbed dwarfism is a cardinal sign which becomes apparent during childhood.

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Orphan drug designation granted to AG-10 for treatment of Transthyretin amyloidosis

On 9 October 2018, U.S. Food and Drug Administration granted 'Orphan Drug' Designation for AG10, a product of Eidos Therapeutics, Inc. (Eidos), for the treatment of Amyloid Transthyretin amyloidosis (ATTR). Jonathan Fox, M.D., Ph.D., president and chief medical officer of Eidos, expressed his deep-dwelled belief that AG10 holds great clinical promise as a disease-modifying drug for these patients.

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Q-CELLS, A PRODUCT OF Q THERAPEUTICS INC., GRANTED ORPHAN DRUG DESIGNATION

In September 2018, the U.S. Food and Drug Administration granted orphan drug designation to Q-Cells for the treatment of transverse myelitis, a rare neurological condition in which the spinal cord is inflamed. Steven Borst, MBA, president and CEO of Q Therapeutics Inc., applauded the move and considered it to be a stellar step toward their goal of advancing glial cell therapy for different types of neurodegenerative diseases.

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Microbiome, the Hidden Treasure of Heath Information

Health is the most treasured commodity ad infinitum. Evolutionary view of human health and disease is inevitable in the face of evidence and time. It is no more a hidden knowledge that out of about 23,000 human gene population, around 37% have homologs in bacteria and 28% originated in unicellular organisms. We are evolved versions of our earlier, pre-evolution existence, which was a unicellular form of life if traced to the origin.

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Male Menopause and Testosterone Crisis

When trying to define roles and personalities of women, Freud wrote that 'Biology is destiny'. A believer in the non-negotiating power of the hormones, Freud perfectly summed up the rationale behind all the wars and fracas that have sabotaged ill-privileged nations and communities and people. Testosterone is the hormone that gives men their masculinity. All the mighty kings and warriors had ample of testosterone in their blood pool to make them dynamic, aggressive and controlling.

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T-Cell Lymphoma: Stories of hope and courage

The tyranny of cancers has slaughtered more minds than bodies. Even the chance mention of a diagnostic possibility clouds the consciousness in a jiffy, demolishing the kernel of life. The mutated molecules of cancer have militated against the body in the most vilifying and varied manners, earning it the reputation of the meanest man-eater disease. The earliest known description of cancer dates back to 3000-28 BC in an ancient Egyptian textbook, Edwin Smith Papyrus, and the document coldly stated of the disease: "There is no treatment1". Even Dinosaurs suffered from cancers and their tumors resembled those of human patients, proving the perpetual existence of cancer. It would not be a hyperbole to state that cancer in humans is as old as the mankind or older even.

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Insulin vs. Glucagon: The relevance of dietary protein

Pancreas is a complex glandular organ and situated intricately and in close association with the liver and the duodenum. Functionally, the organ pancreas is a collaboration of two types of glands (1). The major portion of the pancreas comprises of ‘exocrine’ cells which are involved in the production of digestive enzymes. These enzymes flow into narrow tunes called ducts which merge together to form the single major pancreatic duct. The pancreatic duct opens into the first part of the duodenum (small intestine) and empties out the enzymes.

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How Nano Technology can improve cancer care

Nanotechnology is a marvel of advanced scientific research, virtually providing the possibility of maneuvering things molecule by molecule, or cell by cell. Based on the length scale 'nano,' which is a billionth of a meter, Nanotechnology has arisen to quench our incessant need to miniaturize our technologies and to find exemplary and precise solutions. It has propositioned itself to behave as the ‘Operating System’ of the next generation cancer care.

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