Satellos announced the publication in Nature Communications of new research from a scientific team at the Ottawa Hospital Research Institute (OHRI) that validates the company’s novel approach to treating the underlying cause of Duchenne muscular dystrophy and other diseases. >> Publication available here.
The paper — authored by OHRI researchers, including Satellos co-founder and Chief Discovery Officer Dr. Michael Rudnicki, an OHRI senior scientist and director of the Sprott Centre for Stem Cell Research — reveals that Duchenne muscular dystrophy (DMD, Duchenne) begins during fetal development as a stem cell disease characterized by intrinsic muscle stem cell dysfunction. In the absence of dystrophin, researchers showed that muscle stem cells lose polarity and produce fewer myogenic progenitors, resulting in fewer and smaller muscle fibers. Notably, these changes arise during fetal muscle development before inflammation or tissue degeneration begin to appear.
Importantly, the researchers demonstrated that muscle stem cells lacking dystrophin could be induced to achieve polarity, generate new progenitor cells, and form normal amounts of muscle by blocking the activity of the protein AAK1, findings which support the potential of an AAK1 inhibitor, such as SAT-3247, to restore muscle regeneration in Duchenne.
Unlike treatment approaches in development for Duchenne that focus on helping DMD patients produce a form of dystrophin to help safeguard muscle, Satellos’ differentiated approach aims to restore muscle regeneration for people living with Duchenne through targeted inhibition of AAK1.
“These findings make it abundantly clear that Duchenne begins as a failure of muscle stem cells to build and maintain muscle — without any evidence of myofiber fragility or damage,” said Dr. Rudnicki, senior author of the paper. “By modulating AAK1, we have demonstrated a powerful means to regulate polarity, normalize stem cell function and enhance muscle formation in dystrophic models, pointing to a compelling path toward regenerative treatment strategies.”
Added Frank Gleeson, Satellos co-founder and CEO, “These findings further validate our conviction that correcting stem-cell dysfunction is essential to changing the trajectory of Duchenne. We congratulate Dr. Rudnicki and his OHRI colleagues for uncovering and confirming muscle biology that may open doors to more effective intervention.”
Follow This Page >>> All Clinical Trials for Duchenne
Benimde oğlum dmd kas hastası nasıl yapabilirim bana yardımcı olabilir misiniz
Gülcan Merhaba,
Klinik çalışmalar sayfamızı sürekli takip edin lütfen.
https://dmdwarrior.com/dmd-therapies/