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Accelerating healthcare innovation: the role of Artificial intelligence and digital health technologies in critical path institute’s public‐private partnerships [PDF]

Clinical and Translational Science
Jagdeep T. Podichetty, Sakshi Sardar, Nick Henscheid, Grace V. Lee, J. Rubin Abrams, Wes Anderson, Shu Chin Ma, Klaus Romero   +7 more
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Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease

Clinical and Translational Science, 2021
Interest in drug development for rare diseases has expanded dramatically since the Orphan Drug Act was passed in 1983, with 40% of new drug approvals in 2019 targeting orphan indications. However, limited quantitative understanding of natural history and
Ariana P. Mullin, Diane Corey, Emily C. Turner, Richard Liwski, Daniel Olson, Jackson Burton, Sudhir Sivakumaran, Lynn D. Hudson, Klaus Romero, Diane T. Stephenson, Jane Larkindale   +10 more
doaj   +1 more source

Longitudinal estimated glomerular filtration rate (eGFR) modeling in long‐term renal function to inform clinical trial design in kidney transplantation

Clinical and Translational Science, 2023
Kidney transplantation is the preferred treatment for individuals with end‐stage kidney disease. From a modeling perspective, our understanding of kidney function trajectories after transplantation remains limited.
Luke Kosinski, Eric Frey, Amanda Klein, Inish O'Doherty, Klaus Romero, Mark Stegall, Ilkka Helanterä, Ahmed Osama Gaber, William E. Fitzsimmons, Varun Aggarwal, Transplant Therapeutics Consortium (TTC)   +10 more
doaj   +1 more source

Identifying and characterising sources of variability in digital outcome measures in Parkinson’s disease

npj Digital Medicine, 2022
Smartphones and wearables are widely recognised as the foundation for novel Digital Health Technologies (DHTs) for the clinical assessment of Parkinson’s disease.
George Roussos, Teresa Ruiz Herrero, Derek L. Hill, Ariel V. Dowling, Martijn L. T. M. Müller, Luc J. W. Evers, Jackson Burton, Adrian Derungs, Katherine Fisher, Krishna Praneeth Kilambi, Nitin Mehrotra, Roopal Bhatnagar, Sakshi Sardar, Diane Stephenson, Jamie L. Adams, E. Ray Dorsey, Josh Cosman   +16 more
doaj   +1 more source

Qualifying a Novel Clinical Trial Endpoint (iBOX) Predictive of Long-Term Kidney Transplant Outcomes

Transplant International, 2023
New immunosuppressive therapies that improve long-term graft survival are needed in kidney transplant. Critical Path Institute’s Transplant Therapeutics Consortium received a qualification opinion for the iBOX Scoring System as a novel secondary efficacy
Amanda Klein, Alexandre Loupy, Mark Stegall, Ilkka Helanterä, Luke Kosinski, Eric Frey, Olivier Aubert, Gillian Divard, Kenneth Newell, Herwig-Ulf Meier-Kriesche, Roslyn Mannon, Thomas Dumortier, Varun Aggarwal, Jagdeep T. Podichetty, Inish O’Doherty, Ahmed Osama Gaber, William E. Fitzsimmons   +16 more
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Application of longitudinal item response theory models to modeling Parkinson’s disease progression

CPT: Pharmacometrics & Systems Pharmacology, 2022
The Movement Disorder Society revised version of the Unified Parkinson’s Disease Rating Scale (MDS‐UPDRS) parts 2 and 3 reflect patient‐reported functional impact and clinician‐reported severity of motor signs of Parkinson’s disease (PD), respectively ...
Haotian Zou, Varun Aggarwal, Glenn T. Stebbins, Martijn L. T. M. Müller, Jesse M. Cedarbaum, Anne Pedata, Diane Stephenson, Tanya Simuni, Sheng Luo   +8 more
doaj   +1 more source

Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data

Frontiers in Pharmacology, 2022
The 21st Century Cures Act requires FDA to expand its use of real-world evidence (RWE) to support approval of previously approved drugs for new disease indications and post-marketing study requirements.
Jeffrey S. Barrett, Megan Cala Pane, Timothy Knab, William Roddy, Jack Beusmans, Eric Jordie, Kanwaljit Singh, Jonathan Michael Davis, Klaus Romero, Michael Padula, Bernard Thebaud, Mark Turner   +11 more
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Disease progression joint model predicts time to type 1 diabetes onset: Optimizing future type 1 diabetes prevention studies

CPT: Pharmacometrics & Systems Pharmacology, 2023
Clinical trials seeking type 1 diabetes prevention are challenging in terms of identifying patient populations likely to progress to type 1 diabetes within limited (i.e., short‐term) trial durations. Hence, we sought to improve such efforts by developing
Juan Francisco Morales, Rhoda Muse, Jagdeep T. Podichetty, Jackson Burton, Sarah David, Patrick Lang, Stephan Schmidt, Klaus Romero, Inish O'Doherty, Frank Martin, Martha Campbell‐Thompson, Michael J. Haller, Mark A. Atkinson, Sarah Kim   +13 more
doaj   +1 more source

Development of a model‐based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy

CPT: Pharmacometrics & Systems Pharmacology, 2022
Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X‐linked pediatric disease, have been designed based on the limited understanding of natural disease progression and variability in clinical measures over ...
Karthik Lingineni, Varun Aggarwal, Juan Francisco Morales, Daniela J. Conrado, Diane Corey, Camille Vong, Jackson Burton, Jane Larkindale, Klaus Romero, Stephan Schmidt, Sarah Kim, the Cooperative International Neuromuscular Research Group investigators and Duchenne Regulatory Science Consortium members   +11 more
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Recommendations to Optimize the Use of Volumetric MRI in Huntington's Disease Clinical Trials

Frontiers in Neurology, 2021
Volumetric magnetic resonance imaging (vMRI) has been widely studied in Huntington's disease (HD) and is commonly used to assess treatment effects on brain atrophy in interventional trials.
Kirsi M. Kinnunen, Ariana P. Mullin, Ariana P. Mullin, Dorian Pustina, Emily C. Turner, Jackson Burton, Mark F. Gordon, Rachael I. Scahill, Emily C. Gantman, Simon Noble, Klaus Romero, Nellie Georgiou-Karistianis, Adam J. Schwarz   +12 more
doaj   +1 more source