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Thank you very much @mitoworld.bsky.social for highlighting our work and the upcoming trial! We plan to start recruiting individuals with MT-ATP6 variants later this year.
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Alessandro Prigione
A patient‑derived iPSC screen by Prigione identified sildenafil as a potential therapy for #LeighSyndrome, a devastating #mitochondrialdisease. @cp-cell.bsky.social Now recruiting for clinical trials! mitoworld.org/using-ips-ce... #UMDF #mitoscientists #mitochondria #SynLeigh @ema.europa.eu
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A screen of iPS cells recently identified sildenafil as a potential treatment for Leigh syndrome (LS). Moreover, further testing of the drug showed marked improvement in animal models and human patien...
mitoworld.org
Using iPS Cells to Find Therapies for Mitochondrial Diseases - MitoWorld
www.MitoWorld.org