Treatments for Andersen's Disease

In the previous post, we discussed the possible symptoms of Andersen's Disease. We will now delve into the realm of possibilities with treatments for it.

As mentioned the disease is symptom-specific, thus, treatment for each patient varies. The constant between all patients though is the coordinated efforts of medical professionals, from pediatricians to physicians to cardiologists.

Treatment are better referred to as therapies as there is no cure for Andersen's disease. More funding for research of this disease may one day lead to the possible treatment of Andersen's disease by gene therapy, but until then, therapies directed at Andersen's disease aim to manage the disease.

Management of Andersen's Disease

• Treatment of manifestations
• Prevention of secondary complications
• Surveillance
• Genetic counselling

Treatment of manifestations

Supportive therapies may be directed toward managing the symptoms / effects brought by the disease, for example, cirrhosis, impaired liver function, neuromuscular disease or heart dysfunction. Medications and surgical interventions may be required if cardiomyopathy is involved and heart failure has to be circumvented. In the case of advanced stages of cirrhosis to the liver, a transplant may be required.

Prevention of secondary complications

Dietary measures are taken for patients to maintain normal glucose levels in the blood. This allows for an adequate nutritional intake to improve liver function and muscular strength. 

Surveillance
Assessment of liver, heart and the functions of other organs has tot be checked periodically.

Genetic counselling
Genetic counselling will benefit both the family and the affected individual to reduce the occurance of Andersen's disease in the bloodline.

These approaches are coupled together as the best approach for the long-term management of Andersen's Disease. It no doubt isn't the best, all-in-one solution to cure everything, but this is after all, the only viable solution available currently. The association for glycogen storage diseases (Type 0 to IX, where Type IV is Andersen's Disease) in the United Kingdom welcomes donation to sustain assistance to families affected by Glycogen Storage Disease. Do consider spreading word for the organization to gain more sponsorship and make a donation if possible.

Sources:

http://www.rarediseases.org/rare-disease-information/rare-diseases/byID/394/viewFullReport
http://apbdrf.org/donations/?p=Anti-Sense_-_Recently_Approved_Gene_Therapy