Pompe Disease Emerging Markets

Pompe disease, an inherited rare disease, is often fatal and incapacitates the heart and skeletal muscles. Absence of an essential protein due to the mutation in a gene encoding GAA, leads to a complex buildup of sugar, hence, damaging heart, liver and other organs. The mutated gene hampers the lysosomal-mediated d...

Huntington's disease is an incurable genetic disorder that causes the progressive breakdown of nerve cells in the brain. It is caused by a defect in a gene present on chromosome 4 that causes an excessive build-up of the Huntington protein. This protein build-up ultimately defaces nerve cells in parts of the brain,...

Multiple Sclerosis Multiple Sclerosis is an unpredictable, autoimmune disease that affects the central nervous system. In this disease, the body's own immune system produces cells and proteins (antibodies), which attack the fatty substance,myelin that protects the nerve fibres. So, 30 May 2019 is globally obser...

Before you all start to panic, let us establish one thing first that it is crucial to understand the rising causes behind the rates of Autism and find a solution for it. Every dis-ease or discomfort that affects a human body in any way is without a doubt, a cause of concern. So it is necessary to address it in the ...

The U.S. FDA recently approved the much talked about Novartis’s drug Zolgensma for treating a life-threatening condition Spinal Muscular Atrophy (SMA).  SMA is a neuromuscular disorder that worsens the muscles over time. It happens due to a mutation in the SMN1 gene that results in the deficiency of an SMN pro...

Pulmonary Arterial Hypertension (PAH) is a chronic, life-alarming disease that advances over time. It is a rare disease characterized by the increased blood pressure in the arteries of the lungs. It is somehow different from normal high pressure in a way that blood pressure rises only in the arteries that carry blo...