What are the best treatments for the behavior problems? What are the best treatments for the neuromotor problems? Are these treatments the same, or are they different? An important goal of clinical research is to find new treatments that are useful in Lesch-Nyhan disease.
The simplest method for finding new medications is to get medications from your local doctor, or try them on your own. This is very easy to do, but it is not a very efficient way to find good medications. And some medications could be dangerous. Many people with Lesch-Nyhan disease will waste their time trying the same useless medications as others have tried. Some medications may cause bad side-effects. Because Lesch-Nyhan disease is so rare, few families and few doctors have enough experience to know what should be tried and what should be avoided. Unless there is a central source for information on what worked and what did not, the many experiences of many people all over the world is lost.
The best way to avoid a situation where everyone keeps trying the same things is to perform a clinical trial involving many people with Lesch-Nyhan disease. By counting how many people benefit and how many people get side-effects, it is possible to say if the treatment is really useful. If a new treatment is found to be useful, the information will spread fast. Finding out that a treatment does not work is almost as important as finding out that it does work. If everyone knows that a treatment does not work, then others will not have to try it themselves.
Clinical research involving treatment trials is important to find definitive answers. Many people and families with Lesch-Nyhan disease are suspicious of these trials. Othes are pessimistic and decide the medication is not going to work before they try it. They say "we've tried everything already, and nothing works". But treatment trials cannot succeed without people with Lesch-Nyhan disease helping them. Don't give up in the search for a cure!