C-Path opens access to the Duchenne Regulatory Science Consortium database

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PICTURE: C-Path opens access to the Duchenne Regulatory Science Consortium database to qualified researchers, via its rare disease treatment accelerator, its data and analysis platform. view After

Credit: C-Path

TUCSON, Arizona, April 22, 2021 – Critical Path Institute (C-Path) announced today that it will open access to the Duchenne Regulatory Science Consortium (D-RSC) database to qualified researchers, through its treatment of rare diseases, Data and analysis platform (RDCA-DAP®). The D-RSC database includes data from clinical trials in Duchenne muscular dystrophy (DMD), natural history studies, and clinical data collections. The contributors of these datasets have authorized the sharing of this data externally in order to accelerate the development of therapies for DMD.

DMD is a rare, fatal, genetic neuromuscular disease diagnosed in childhood and primarily affecting men. It causes progressive muscle loss, leading to loss of the ability to walk, loss of upper body strength, progressive breathing problems, cardiomyopathy, and premature death. While there are medical treatments that can help slow its progression, there is currently no cure for DMD.

C-Path’s RDCA-DAP provides a centralized and standardized infrastructure to support and accelerate the characterization of rare diseases, with the aim of accelerating the development of therapies for all rare diseases. By sharing this valuable data resource through RDCA-DAP, D-RSC aims to extend and amplify the availability of data to accelerate the development of therapy for DMD, foster the development of tools to accelerate clinical trials for DMD and help develop a deeper understanding of natural DMD. the story.

“People with Duchenne urgently need effective treatments,” explained C-Path Scientific Director Klaus Romero, MD, MS, FCP. and making the data available to qualified researchers will have a profound effect on speeding up the drug development process. “

All data in the D-RSC database has been mapped to the current structures of the Clinical Data Interchange Standards Consortium (CDISC) Standard Data Tabulation Model (SDTM). The data in the D-RSC database is fully anonymized and the datasets are integrated, so researchers will not be able to identify study participants and the data can be used in an aggregate fashion.

D-RSC was formed through collaboration between the Critical Path Institute and the Parent Project Muscular Dystrophy. Individuals and companies from industry and academia are members of the consortium, and representatives from the United States Food and Drug Administration, the European Medicines Agency, and the National Institutes of Health have joined the committee. coordination of the consortium as observers.

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Researchers interested in providing data to D-RSC and / or using the database to advance DMD-related research initiatives can learn more about: https: //path c.org /programs /d-rsc /Overview/database access / or contact D-RSC Associate Director Ramona Belfioire-Oshan, Ph.D., at [email protected]

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the United States Department of Health and Human Services (HHS) and is funded 55% by the FDA / HHS, for a total amount of $ 14,575,306, and 45 % from non-governmental sources. ), totaling $ 11,916,747. The content is that of the authors and does not necessarily represent official opinions or an endorsement by the FDA / HHS or the US government. For more information, please visit FDA.gov.

Critical Path Institute (C-Path) is an independent, non-profit organization established in 2005 as a public-private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, thereby accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, universities, patient organizations, disease foundations and dozens of pharmaceutical and biotechnology companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff at several other locations. For more information, visit c-path.org and c-path.eu.

Media contact:

Kissy Black

Path C

615.310.1894

[email protected]

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