Lysosomal Storage Diseases Therapeutics are getting better with continuous R&D activities among key pharmaceutical companies

 

Lysosomal Storage Disease is a medical condition where proteins, usually DNA, become deformed or lose their catalytic ability. The human genetic material, which is called DNA, is a highly dynamic and sophisticated part of our system. Due to a process called mutations, DNA becomes altered and either changes its directions or becomes a different size. In either case, the genetic information is then unreadable by cells known as enzymes. There are various types of lysosomal storage diseases including Gaucher disease, Fabry disease, Pompe disease, Niemann-Pick disease, Tay-Sachs disease, etc.

Lysosomal storage diseases therapeutics aim at correcting these mutations so that the DNA can be read again by cells. Since the function of the human body is to produce new cells, any change in DNA is extremely dangerous. People with lysts have had to live with the lifelong threat of illness because even minor changes in their DNA structure could lead to serious illness. The human population as a whole has always lived by eating fresh fruits and vegetables, which are rich in essential nutrients and vitamins, and minimizing caloric intake through diet.

Due to this, diseases such as diabetes, cancer, and heart disease have tended to increase over the years. A deficiency in vitamin D, for instance, has been linked to increased cancer risks, which is precisely the case for those with Lysosomal Storage Diseases. People with the disease can no longer absorb the nutrients from foods because the proteins have become rearranged, sometimes becoming pairs and disjointed. These changes often occur without any obvious symptoms. However, prolonged exposure to certain conditions, such as excessive heat or chemicals, can cause certain symptoms to arise. Symptoms can range from swelling of the ankles to liver disease and kidney damage. Recently, in August 2020, Centogene N.V., a Germany-based company, collaborated with Evotec for Gaucher disease drug discovery.