1. Translation, publication

Translation and publication of foreign language documents for the benefit of the Hungarian community. Monitoring press releases and research advances, publishing interim and final results and analyses.

Hungarian families facing the devastating diagnosis of Duchenne Muscular Dystrophy (DMD) have very limited access to information about the disease other than that the condition is incurable, that their child will be wheelchair-bound by the age of 10, and that he will most likely not live past his 20th birthday. This despite the fact that, thanks to patient organizations and researchers worldwide, there is a great deal of information available e.g. in English and German, both on the standards of care and on the latest advances in the search for a cure.

1. a) Translating documents

Our Foundation aims to fill this gap in the availability of information. One of our ongoing projects involves making foreign language materials accessible to Hungarian families affected by DMD.

The translated documents will provide the families with detailed information about the disease, as well as about current and prospective therapeutic possibilities. Furthermore, disseminating the standards of care will help the organization TREAT-NMD in its effort to spread their guidelines in Hungary, a recent addition to the list of member countries.

2. b) Press monitoring

Our Foundation monitors press releases, keeping track of up-to-date information on research, clinical trials, plans and results directly connected to DMD, as well as basic research achievements that could be relevant to future treatment approaches or a better and faster diagnosis of the condition.

The results are published on our website as online news and comprehensive reports, as well as in the form of printed publications to be distributed at various events organized for the benefit of patients and their families.

2. TCM research

Researching the results of Traditional Chinese Medicine (TCM) in treating muscular dystrophies, concentrating mainly on Chinese medical facilities.

In the Far-East, especially China, Traditional Chinese Medicine is successfully combined with western medical approaches to treat various conditions. Chinese doctors are often successful in cases where western medicine fails to present results. The Foundation will survey Chinese facilities (universities, clinics and hospitals) with promising achievements in conjoining TCM and western medicine. Part of the research will involve sending a Hungarian DMD patient to China and funding his treatment there in order to gain first hand experience. The aim of the research is to assemble an informative list in which facilities are ranked according to efficiency, achievements, length and price of treatment, also taking into account the age of patients admitted to each facility. The current practice of Hungarian parents is to decide on a facility in an ad hoc manner, often taking their children to unsupervised treatments like stem cell therapies of uncertain backgrounds, in which case the risks outweigh the benefits by far. As a result of the research, Hungarian DMD patients will gain access to competent doctors working under supervised institutions. Due to the size of the country and the number of institutions to be vetted, we plan to carry out the research with the help of a Chinese consulting firm.

3. Symposium

Organizing a series of small conferences with the aim of establishing and publishing an expert opinion on the combination and dosage of nutritional supplements to be taken by children suffering from Duchenne Muscular Dystrophy.

International patient organizations and doctors worldwide recommend a wide range of nutritional products to patients in order to help delay the loss of muscle function. With the help of a group of experts, our Foundation plans to synchronize and possibly amend these recommendations, as well as establish the weight and condition dependant dosage for every age group. The expert group we have assembled for this purpose consists of dietitians, neurologists and pharmacists.

4. Creating and maintaining a patient database

Duchenne Muscular Dystrophy is caused by the lack or dysfunctionality of dystrophin, a vital protein found in muscles. The root of this problem is a mutation in the genetic code of this protein. The dystrophin gene is the largest of all the human genes, thus the mutations can be of various types and located at different sections of the gene. Experimental therapeutic approaches currently in the works usually focus on a particular type or locus of mutation, and therefore each approach concerns only a small, well-defined group of patients. This makes it exceedingly important to maintain accurate and up-to-date information on DMD patients

5. Japanese grant

Offering a research grant focusing on the efficiency of methods used to treat DMD in Japan.

According to statistics, life expectancy of DMD patients in Hungary is exceeded not only in Western Europe, but even more so in Japan, where this figure is probably the highest in the world. Our Foundation plans to extend a grant to a PhD student or a researcher wishing to investigate the methods used in Japan to treat DMD at a Japanese university or research center. We hope that this research will shed light on the (currently mysterious) reasons for this difference in life expectancy. Furthermore, the result could complement the efforts of TREAT-NMD in establishing comprehensive standards of care for DMD patients based on experience gathered in Europe and the US.

In order to minimize costs, we plan to fund the research project in coordination with one of the research grants currently available in Japan.