What are the different types of Tay-Sachs treatments?
Tay-Sachs disease is a very rare genetic disorder that is usually impossible to treat or treat. Almost all infants who acquire this disease have fatal complications before the age of four and older patients who develop symptoms have also drastically reduced the range of life. Most of the current Tay-Sachs are a supportive nature: they are designed to relieve pain and other symptoms and help patients remain as comfortable as possible, as possible, as the disease gradually deteriorates. The ongoing genetic and pharmaceutical research has a promise in the near future to create effective Tay-Sachs therapies, although many other studies are needed than specific therapies are considered safe. There is evidence that suggests that stem cell transplantation, registration of enzymes and gene therapy can lead to a reliable Tay-Sachs drug.
Tay-Sachs patients have a defective hex-a genes. Failing to produce a functional version of a particular type of enzyme called BETA-Hexosaminidase and (BHA). Without BHA, nerve cells in the brain and spinal cord are unable to break down oily bearings known as gangliosides, leading to progressive damage to nervous and cellular death. Because there are no proven medicines or therapy to improve the functioning of BHA, the current treatment of Tay-Sachs is aimed at fighting the various symptoms that the disease causes.
Beauty are a relatively common complication of Tay-Sachs, so anticonvulsive drugs are often prescribed to reduce their severity and frequency. Other medicines can be administered in combating problems with blood pressure, depression, poor muscle function and respiratory infections. Patients who have problems and breathing problems must have oxygen therapy and be equipped with stomach tubes to ensure that they receive proper nutrition. Adolescents and adults who develop Tay-Sachs are often scheduled for physical therapy to help themOhli keep the muscles SA, despite their disabilities, learn how to stay mobile. Despite the great efforts to support and delay the onset of neurological problems, patients eventually succumb to the disease.
Currently, several theories are being studied that can lead to a successful Tay-Sachs treatment in the future. One experimental idea claims that defective beta-hexosaminidase and enzymes can be replaced by healthy endy in the patient's blood. Enzyme substitution therapy is a well-established treatment of some genetic diseases that affect the lungs, hearts or other organs of the body, but Tay-Sachs represents unique challenges because enzyme deficiencies affect the brain. There is not yet an effective way to get healthy enzymes through the blood -brain barrier.
Other experimental treatments Tay-Sachs include gene therapy and stem cell transplantation. Gene therapy includes the introduction of a large amount of hex-a genes into the patient's system in the hope that its DNA incorpaciouslyThey are echoing. Stem cells harvested from a donor bone marrow or umbilical blood blood may also be beneficial for patients with Tay-Sachs. Healthy stem cells that can properly encode Hex-A Gen can be able to increase the levels of enzyme in the brain. In continuing testing, many doctors are convinced that the disease will be curable one day.