Our research project has just started having the goal to stop/slow progression of Duchenne/Becker Muscular Dystrophy.

The project is headquartered in Brazil at a Genetic Therapy Center (CTG) in Ribeirao Preto, about 250 miles from São Paulo. This research center is affiliated with Association of Friends of DMD (AADM) and is already setup to study drugs in mdx mice and golden retriever dogs. The head of AADM and CTG is Edna Maria Pupin. Edna is the mother of Murilo, 25 year old man with DMD. She is also the "mother and heart" of this Fight in Brazil and to this research center (CTG). Dr. David Feder who you all know is the Principal Investigator. Noted and knowledgeable experts from Germany, France, England and the U.S. have already agreed to serve on our advisory board and will offer their advice and consent.

CTG was initially constructed and equipment was supplied using funds contributed by parents and the State of São Paulo Research Foundation (Fapesp). This centers main and only goal is to study muscular dystrophy. The University of Ribeirao Preto is the principal supervising university but other universities such as the University of Sao Paulo will be involved as the project becomes larger. At present, parents from Brazil have made contributions to keep this project going.

It is difficult to raise this level of funding but we want to keep this project going while the money is being raised. Please send your contributions directly to Ms. Pupin at AADM. The address is:

Edna Maria Pupin

Av. Bandeirantes, 2419

Vila Virginia, Ribeirao Preto, SP, Brazil
14030-670

e.mail: [email protected]

Meanwhile Dr. Pelofsky will continue his efforts to raise the entire sum.

The drugs that will be investigated during this effort are already on the market and already approved by the U.S. FDA (Food and Drug Administration) but for other applications. The basic reason is that the pharmaceutical companies absolutely refuse to petition the U.S. Federal Drug Administration (FDA) for diseases other than those already approved by FDA for that particular drug. Because these drugs are on the market there is a wealth of information for each of them.

Each drug that will be tested has certain properties that we feel will help alleviate the symptoms of this terrible disease. These drugs will first be tested in mdx mice and GRMD (Golden Retriver muscular dystrophy). The pertinent pharmaceutical companies have already completed safety studies on such animals but refuse to divulge their data to us. If the animal testing shows positive results, conduct clinical trial studies under the laws and guidelenes of Brazil will be initiated in clinics around the world that will agree and sanction our protocol.

See photos:

Since the early 1990s, steroids have been used to delay the progression of the disease but there are many adverse side effects. It is our contention that if steroids show efficacy then there must be other drugs that would provide successful treatments without the adverse side effects of steroids. The following are fruitful areas of research:

• Mitochondrial stabilizing/enhancing compounds;
• Plasma membrane stabilizers;
• Calcium reduction agents;
• Fibrosis reducing agents; and
• Agents that oxygenate affected skeletal muscle tissues.

We will post all information; good or bad, as the project goes forward. Names of researchers, names of researchers in the advisory board, names of drugs, how this project is financed will be published in the future. We will of course let you all know when our new site is up and running.

On behalf of Fight for a Future

Berit