Post by David Feder on Jul 31, 2009 18:02:07 GMT -5
World Muscle Society Annual Meeting - Abstracts
Parents vision on Duchenne muscular dystrophy (DMD) research
D. Feder1 and B.S. Segtnan2
1ABC Faculty of Medicine, Pharmacology, Sao Paulo, Brazil
2Fight For a Future, Director, Orkdal, Norway
Available online 30 July 2009.
Despite incredible advances in knowledge on muscle physiology and diseases, DMD remains as an incurable disease. The discovery, 20 years ago, of the involved gene, had leaded to the believing of a fast cure for the disease. Gene therapy remains as an efficient procedure in animal models but is still experimental in humans. Advances in the life-quality and survival time were obtained with steroids, heart protection and noninvasive ventilation. Groups of parents of DMD patients are among the most involved people in the research financing. Every year large sums of money are invested in research that has not brought short-term results. In other words, parents and DMD patients are contributing members of a “club” without wanting to be and whose benefits they may not enjoy. Groups of parents experience the feeling that the current generation was abandoned. The results obtained in animals had not lead to clinical trials, then, parents begin to use drugs before the performance of those studies. Clinical trials on new drugs, when conducted, take many years to be carried out, and patients on death risk or on risk of developing severe complications are excluded of them. Therefore, these patients will not be able to use these drugs.. A growing number of patients have been using drugs as losartan, protandim, etc. without medical prescriptions. Another consequence of this delay is parents organizing their own “clinical trials”. Therefore, parents have some suggestions concerning to this situation:
(a) multicenter studies with a drug thingytail.
(b) clinical studies on nonambulatory patients trying to postpone DMD complications.
(c) Use of drugs or therapies in patients on death risk or in those seriously ill.
(d) researchers must take information on trials carried out by parents, monitoring those trials and work in colaboration with parents.
(e) specifically-destined profits for the research that could bring immediate results for the current generation of patients.
Parents vision on Duchenne muscular dystrophy (DMD) research
D. Feder1 and B.S. Segtnan2
1ABC Faculty of Medicine, Pharmacology, Sao Paulo, Brazil
2Fight For a Future, Director, Orkdal, Norway
Available online 30 July 2009.
Despite incredible advances in knowledge on muscle physiology and diseases, DMD remains as an incurable disease. The discovery, 20 years ago, of the involved gene, had leaded to the believing of a fast cure for the disease. Gene therapy remains as an efficient procedure in animal models but is still experimental in humans. Advances in the life-quality and survival time were obtained with steroids, heart protection and noninvasive ventilation. Groups of parents of DMD patients are among the most involved people in the research financing. Every year large sums of money are invested in research that has not brought short-term results. In other words, parents and DMD patients are contributing members of a “club” without wanting to be and whose benefits they may not enjoy. Groups of parents experience the feeling that the current generation was abandoned. The results obtained in animals had not lead to clinical trials, then, parents begin to use drugs before the performance of those studies. Clinical trials on new drugs, when conducted, take many years to be carried out, and patients on death risk or on risk of developing severe complications are excluded of them. Therefore, these patients will not be able to use these drugs.. A growing number of patients have been using drugs as losartan, protandim, etc. without medical prescriptions. Another consequence of this delay is parents organizing their own “clinical trials”. Therefore, parents have some suggestions concerning to this situation:
(a) multicenter studies with a drug thingytail.
(b) clinical studies on nonambulatory patients trying to postpone DMD complications.
(c) Use of drugs or therapies in patients on death risk or in those seriously ill.
(d) researchers must take information on trials carried out by parents, monitoring those trials and work in colaboration with parents.
(e) specifically-destined profits for the research that could bring immediate results for the current generation of patients.