Can you discuss, for any one of the 4 transient treatment approaches, how you might modify the treatment protocol to come closer to what I suspect is our ideal goal, a one-step, permanent “cure”?
To address some of the key challenges among patients with congenital muscular dystrophy type Ullrich with an aim of developing temporary treatment approaches, doctors need to appreciate complication that characterizes the life experience of the patients. In reference to Bolduc paper and Shen’s publications, I have highlighted these challenges that are key in defining the modifications of treatment protocols.
First, living with a genetic disease means a lifetime experience of treatment and medical attention, an experience that mostly characterizes the lives of patients with congenital muscular dystrophy type Ullrich (UCMD). The condition is common among children in their early childhood stages and is characterized by severe disorders that arise from weakened muscles. Bolduc paper appreciates that the condition does not have permanent treatments, a position that poses a significant challenge to the health of the patients.
Another equally great concern among the patients is the financial obligation that is associated with management of the life long condition. UCMD is also significant reliant on the use of respiratory support systems especially during night-times, and in severe cases, the continuous subjection to Mechanically supported ventilation may expose the patient to ventilator acquired pneumonia (VAP) among their related cases. VAP is a condition that is common among patient in the Intensive care Unit (ICU) with mechanically supported ventilation and is commonly associated with poor oral hygiene. Shen appreciates that VAP increases mortality among children.
Recurrent lower respiratory tract infections are one of the most common experiences that are common among patient with weakened immune systems. The weakened muscular activity that, in server cases, necessitates mechanical ventilation support that exposes the child to respiratory tract infection that is best-averted form a stable immune system.
Transient treatment approaches of treatment modifications
Bolduc publication on health appreciates that research on Ullrich congenital muscular dystrophy has been advanced to address some of the key challenges facing the patient especially the lifetime management of the condition.
- The leading concern among researchers is the management of the condition and ensuring that the patient experience an average healthy lifestyle. Some measures like surgery have since been recommended for cases like contractures and scoliosis. Bolduc paper acknowledges that some patients suffering from Ullrich congenital muscular dystrophy significantly benefit from cyclosporin A.
- Administration of antibiotics manages the respiratory tract related condition that builds up to VAP in ICUs. Gastronomy is also a technique that has also been recommended.
- The various health insurance institutions have been advised for patients. Bolduc paper earlier indicated the enormous financial burden associated with chronic diseases highlighting the need for proper measures to mitigate the financial inconvenience of the condition.
- Maintenance of optimum oral hygiene is also a measure that has been emphasized. Oral health, including the use of tooth, brushed as well as appropriate chemicals, aid in preventing the onset of VAP among patients under mechanical ventilation.
- Other recommended measures of managing mechanically ventilated patients suffering from severe cases of Ullrich congenital muscular dystrophy includes prevention of gastric relaxes, proper equipment maintenance, avoiding cross contamination and proper air way management.