Fact 8: The Battle Against Gaucher’s Disease – Treatment Approaches
The fight against Gaucher’s Disease takes various forms, each tailored to meet the specific needs of the patient. The primary goal of treatment is to manage symptoms, improve the quality of life, and extend the lifespan.
One of the primary treatments for Gaucher’s Disease is enzyme replacement therapy (ERT). This approach involves providing the patient with the deficient enzyme, glucocerebrosidase, artificially synthesized. ERT has been successful in reducing the size of enlarged organs, increasing blood cell counts, and improving bone symptoms.
Substrate reduction therapy (SRT) is another treatment approach that works differently than ERT. Instead of replacing the deficient enzyme, SRT reduces the production of the fatty substance that the enzyme would normally break down, thereby reducing its accumulation in cells.
Additional treatment options may include supportive therapies to manage symptoms such as pain, anemia, or low platelet counts. This can involve the use of pain medications, blood transfusions, or other interventions as required.
While current treatments for Gaucher’s Disease can manage symptoms, they are not a cure. However, with ongoing advancements in medical research, the hope for more effective treatments and eventually, a cure, remains steadfast. (8)
