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We continue to advance in the research of Limb-girdle Muscular Dystrophy 1F/D2
Advances in research after the 8th International Congress on our Muscular Dystrophy
When will we be able to stop Limb-girdle Muscular Dystrophy 1F/D2 (LGMDD2) and improve its symptoms? We don’t have an exact date. Although we are getting closer every day. Thanks to the collaboration of hundreds of people who, like you, continue to trust in research, we are getting closer to finding a medicine.
Last Thursday, October 31, we met with all the research teams involved to celebrate the 8th International Congress of LGMDD2 and learn the details of this work, as well as coordinate the next steps. Sharing advances, barriers, discoveries and ideas accelerates the possibility of being able to take the long-awaited drug that stops our disease, as well as finding a definitive cure.
In 2025, the Conquistando Escalones Association will be 10 years old and the objective is clear: to get enough money so that, when we are told that we can proceed with the clinical trial of the first drug, we can carry it out. We will certainly need a minimum of €100,000 to carry it out. We are already preparing various initiatives to continue moving forward in this very marked year thanks to your support and the work of the researchers. The financial and research goal is ambitious, but together we can achieve it.
More details on the advances in research
Although in many cases they are preliminary results or paths in progress, we leave you with some of the key brushstrokes in terms of news about the research of our disease that the congress has left:
- The team of Prof. Artero from the University of Valencia and Incliva presented new avenues of gene therapy (beyond the work on gene editing by Crispr which is also being worked on) with which they seek to silence the defective gene that causes our disease. This is done using antisense oligonucleotides and they are evaluating their effects and will continue to delve deeper into it. At the same time, among other things, they continue to focus on developing an effective model to be able to test medicines and confirm the results already obtained in cells, as required by the State Agency for Medicines as a final step before clinical trials in humans.
- Dr. Cenacchi’s team from the University of Bologna has presented the novelty of the muscular structures they have created in 3D, which better reproduce muscular functionality. This is thanks to a 3D bioprinter that makes these samples and the advantages it offers are, among others, greater speed in progress, reduced costs and a better representation of the complexity of tissues and muscles. Thanks to this, they have already managed to see significant differences in myogenesis that were appreciated in 2D, which can substantially help to better understand our disease and how our genetic alteration acts.
- Dr. Alcamí’s team from the Carlos III Institute in Madrid has conducted a very complex and interesting study, evaluating the overexpression of proteins in our Muscular Dystrophy, which opens up possible new therapeutic targets for its treatment and new clues to better understand our disease and potential new medications. In addition, they continue with the investigations of the interaction of our disease with HIV and the search for treatments that help both ailments.
- Dr. Vílchez, for his part, presented the advances in the investigation of a muscular disease different from the one we suffer from, which surprisingly has very similar characteristics to ours, which may confirm some theories that were already being considered about LGMDD2 and possible ways to stop it.
The laboratories continue to work on these and other fronts, of which, as usual, we will keep you informed in detail in upcoming communications, as well as sharing any scientific publication that is made on the findings that are achieved. We will also tell you shortly about everything that we are preparing on the occasion of our tenth anniversary. Thank you very much for your help.