c-percorso
January 2024: A new C-Path Task Force Aimed at Accelerating Drug Development for Mitochondrial and Metabolic Diseases is launched!
This new task force will allow our community to collaborate, share data, and accelerate development of treatments with the support of C-Path and their Rare Disease Cures
Accelerator-Data and Analytics Platform (RDCA-DAP ® ). RDCA-DAP is an FDA-funded platform that creates the collaborative, non-competitive space to share existing patient-level data and encourages the standardization of new data collection.
Piattaforma di dati e analisi dell'acceleratore di cure per le malattie rare
RDCA-DAP è un'iniziativa finanziata dalla FDA che fornirà un'infrastruttura centralizzata e standardizzata per supportare e accelerare la caratterizzazione delle malattie rare con l'obiettivo di accelerare lo sviluppo della terapia.
Critical Path Institute (C-Path) and Cure Mito Foundation (Cure Mito) today announced a joint collaboration to significantly promote data sharing and accelerate Leigh syndrome and other rare mitochondrial disease data incorporation into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®).
Leigh syndrome, also known as Leigh disease, is a Mitochondrial disorder that affects about 1 out of 77,000 live births, with symptoms often appearing within the first year of life. It is a debilitating neurological disorder that can affect the development of mobility, posture and mental capacities in children, with these capabilities occasionally being lost after a period of seemingly normal growth. Though unusual, symptoms may not show until the early adult years, while others may show gradual signs throughout infancy.
“Progress towards the establishment of approved therapies for people with Leigh syndrome and other rare mitochondrial diseases is reliant on the participation of organizations like the Cure Mito Foundation,” said RDCA-DAP Scientific Director Alexandre Betourne, Ph.D., Pharm.D. “This is a laudable step in that direction, and we are thrilled to be partnering with Cure Mito to promote data sharing, standardization and develop tools that will accelerate the development of treatments for Leigh syndrome and other mitochondrial disorders.”
“Patients enroll in registries and research studies hoping that their participation will matter,” said Sophia Zilber, Cure Mito board member. “Cure Mito Foundation recognizes this and is committed to advancing the way data is collected, accessed and used, and empowering the patient community to be informed and active contributors of their data to advance research, treatments and eventually cures. We are proud to partner with C-Path in these important and much needed efforts.”