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.
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