The notices it replaced
- Reissues: PAR-23-159 Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
- 2 steps back: PAR-22-101 Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
- 3 steps back: PAR-18-953 Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Who won it before
46 competing awards (new and renewals) from FY2014 to FY2025 under this notice and the notices it replaced: $12,630,105 in first-year total cost, median $243,950.
Most frequent study sections
- Special Emphasis Panel[ZTR1 RD-3 (01)]19
- Special Emphasis Panel[ZTR1 RD-7 (01)]15
- Special Emphasis Panel[ZTR1 RD-3 (02)]4
- Special Emphasis Panel[ZTR1 RD-5 (01)]4
- Special Emphasis Panel[ZTR1 RD-2 (01)]3
Most frequent institutions
- CHILDREN'S HOSP OF PHILADELPHIA6
- DUKE UNIVERSITY3
- UNIVERSITY OF CALIFORNIA, SAN DIEGO3
- JOHNS HOPKINS UNIVERSITY2
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO2
| FY | Project | PIs | Institution | First-year cost | Study section |
|---|---|---|---|---|---|
| 2025 | Patient-Centric Clinical Outcome Measures for Multiple Sulfatase Deficiency (MSD)1R21TR006299-01A1 · under PAR-23-159 | ADANG, LAURA ANN | CHILDREN'S HOSP OF PHILADELPHIA, PA | $489,500 | ZTR1 |
| 2025 | Validating interferon score and phospho-STING level as disease biomarkers of COPA syndrome1R21TR005838-01 · under PAR-23-159 | SHUM, ANTHONY | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, CA | $462,188 | ZTR1 |
| 2025 | Natural History Study and Identification of Patient-Centric Clinical Outcome Assessments in TUBB4A-LD (NIH R21)1R21HD120719-01 · under PAR-23-159 | SEVAGAMOORTHY, ANJANA | CHILDREN'S HOSP OF PHILADELPHIA, PA | $415,245 | ZTR1 |
| 2025 | Trial readiness for LCAT deficiency1R21TR005433-01 · under PAR-23-159 | CUCHEL, MARINA | UNIVERSITY OF PENNSYLVANIA, PA | $401,566 | ZTR1 |
| 2025 | 4-hydroxymandelate for HPDL deficiency, a novel mitochondrial encephalopathy1R21TR006111-01A1 · under PAR-23-159 | KIM-MCMANUS, OLIVIAYANG, JENNIFER HUASI | UNIVERSITY OF CALIFORNIA, SAN DIEGO, CA | $398,750 | ZTR1 |
| 2025 | Assessment of restoration of an internally deleted dystrophin as a surrogate biomarker for future clinical trials of a gene editing therapy in Duchenne muscular dystrophy1R21HD117763-01 · under PAR-23-159 | YOUNG, COURTNEY S | MYOGENE BIO, LLC, CA | $302,500 | ZTR1 |
| 2024 | A Clinical Trial Readiness Study of Patient Reported Outcome Measures in Thrombotic Thrombocytopenic Purpura (TTP)1R21TR004694-01A1 · under PAR-23-159 | COLES, THERESAONWUEMENE, OLUWATOYOSI ADEFUNKE | DUKE UNIVERSITY, NC | $456,348 | ZTR1 |
| 2024 | Triangulating outcome assessments for blepharospasm1R21TR005231-01 · under PAR-23-159 | PETERSON, DAVID A | UNIVERSITY OF CALIFORNIA, SAN DIEGO, CA | $450,836 | ZTR1 |
| 2024 | Clinical Trial Readiness for Posterior Cortical Atrophy Syndrome1R21TR004695-01A1 · under PAR-23-159 | PELAK, VICTORIA SUSAN | UNIVERSITY OF COLORADO DENVER, CO | $429,000 | ZTR1 |
| 2024 | AI/ML and iPSC-Derived Organoids for Myotonic Dystrophy Drug Discovery1R21TR004938-01 · under PAR-23-159 | PANG, PAUL | GREENSTONE BIOSCIENCES, INC., CA | $110,000 | ZTR1 |
| 2023 | Plasma neurofilament light chain as a potential disease monitoring biomarker in Wolfram syndrome1R21HD112910-01 · under PAR-22-101 | HERSHEY, TAMARA G | WASHINGTON UNIVERSITY, MO | $427,625 | ZTR1 |
| 2023 | Clinical Trial Readiness for Monitoring Muscle Inflammation in Duchenne Muscular Dystrophy1R21TR004895-01A1 · under PAR-22-101 | HOFFMAN, ERIC P. | STATE UNIVERSITY OF NY,BINGHAMTON, NY | $237,360 | ZTR1 |
| 2023 | Synergistic clinical outcome assessments for cervical dystonia1R21TR004422-01 · under PAR-22-101 | PETERSON, DAVID A | UNIVERSITY OF CALIFORNIA, SAN DIEGO, CA | $206,665 | ZTR1 |
| 2022 | Brain MRI to pre-symptomatically predict seizure onset for Sturge-Weber Syndrome1R21TR004265-01 · under PAR-18-953 | COMI, ANNE MOU, YANGMINGPINTO, ANNA LECTICIA | BOSTON CHILDREN'S HOSPITAL, MA | $271,133 | ZTR1 |
| 2022 | Functional Biomarkers for ALS1R21TR003589-01A1 · under PAR-18-953 | WILKINS, HEATHER M. | UNIVERSITY OF KANSAS MEDICAL CENTER, KS | $253,794 | ZTR1 |
| 2022 | Menstrual Cycle-Related Symptom Variability as a Prognostic Indicator in Lymphangioleiomyomatosis1R21TR003858-01A1 · under PAR-18-953 | GUPTA, NISHANT | UNIVERSITY OF CINCINNATI, OH | $243,000 | ZTR1 |
| 2022 | Validating absolute lymphocyte count and plasma sphingosine-1-phosphate as disease biomarkers of sphingosine phosphate lyase insufficiency syndrome in anticipation of a pyridoxine clinical trial1R21TR004262-01 · under PAR-18-953 | SABA, JULIE D | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, CA | $242,250 | ZTR1 |
| 2022 | A Novel Therapeutic Approach to Treat Focal Segmental Glomerulosclerosis (FSGS)1R21TR004257-01 · under PAR-18-953 | SPURNEY, ROBERT | DUKE UNIVERSITY, NC | $241,500 | ZTR1 |
| 2022 | Disease Severity Stratification in Multiple Sulfatase Deficiency1R21HD106348-01A1 · under PAR-18-953 | AHRENS-NICKLAS, REBECCA CLARE | CHILDREN'S HOSP OF PHILADELPHIA, PA | $232,535 | ZTR1 |
| 2022 | REFINING METRICS OF FOOD BEHAVIOR IN SMITH-MAGENIS SYNDROME1R21HD106345-01A1 · under PAR-18-953 | ELSEA, SARAH H.SISLEY, STEPHANIE RENEE | BAYLOR COLLEGE OF MEDICINE, TX | $225,420 | ZTR1 |
| 2022 | Clinical Trial Readiness - Primary Ciliary Dyskinesia (CTR-PCD)1R21TR004057-01 · under PAR-18-953 | ROSENFELD, MARGARET | SEATTLE CHILDREN'S HOSPITAL, WA | $212,161 | ZTR1 |
| 2022 | Cell-free DNA-Based Analysis for Diagnosis, Monitoring and Optimization of Therapy for Patients with Primary Central Nervous System Lymphomas1R21TR004059-01 · under PAR-18-953 | BETTEGOWDA, CHETANHOLDHOFF, MATTHIAS | JOHNS HOPKINS UNIVERSITY, MD | $204,687 | ZTR1 |
| 2021 | Targeting FOXM1 in chemo-resistant monocytic AML1R21TR003587-01 · under PAR-18-953 | GARTEL, ANDREI L | UNIVERSITY OF ILLINOIS AT CHICAGO, IL | $263,793 | ZTR1 |
| 2021 | PAR-18-953 (ASCOT-DMD)1R21TR004007-01A1 · under PAR-18-953 | THANGARAJH, MATHULA | VIRGINIA COMMONWEALTH UNIVERSITY, VA | $256,214 | ZTR1 |
| 2021 | MRI Biomarkers of Disease Progression in Inherited Neuropathies1R21TR003312-01A1 · under PAR-18-953 | DORTCH, RICHARDLI, JUN | WAYNE STATE UNIVERSITY, MI | $246,172 | ZTR1 |
| 2021 | Dose Optimization of Nervonic Acid - a Potential Therapy to Alleviate Disease Progression in Adrenoleukodystrophy1R21TR003941-01 · under PAR-18-953 | KARTHA, REENA V | UNIVERSITY OF MINNESOTA, MN | $232,500 | ZTR1 |
| 2021 | Defining ichthyosis in Sjogren-Larsson syndrome for clinical trial preparedness1R21HD106350-01 · under PAR-18-953 | RIZZO, WILLIAM B. | UNIVERSITY OF NEBRASKA MEDICAL CENTER, NE | $223,198 | ZTR1 |
| 2021 | Clinical validation of at-home methods of sleep assessment in Rett Syndrome1R21TR003942-01 · under PAR-18-953 | PETERS, SARIKA U | VANDERBILT UNIVERSITY MEDICAL CENTER, TN | $214,422 | ZTR1 |
| 2021 | Biomarkers for 12-lipoxygenase inhibition as a therapeutic intervention for heparin-induced thrombocytopenia and thrombosis (HIT/T)1R21TR003185-01A1 · under PAR-18-953 | HOLINSTAT, MICHAEL ALLAN | UNIVERSITY OF MICHIGAN AT ANN ARBOR, MI | $208,600 | ZTR1 |
| 2021 | Optimizing Muscular Dystrophy Clinical Trial Designs using Modeling and Simulation1R21TR004006-01 · under PAR-18-953 | KIM, SARAH | UNIVERSITY OF FLORIDA, FL | $190,625 | ZTR1 |
| 2020 | Development of a reliable, valid, and sensitive outcome measure in Rett syndrome1R21HD103348-01 · under PAR-18-953 | NEUL, JEFFREY L | VANDERBILT UNIVERSITY MEDICAL CENTER, TN | $275,091 | ZTR1 |
| 2020 | Characterization of RASopathy Mutations Underlying Lymphatic Anomalies and Preparation for Clinical Development1R21TR003331-01 · under PAR-18-953 | LI, DONG | CHILDREN'S HOSP OF PHILADELPHIA, PA | $264,000 | ZTR1 |
| 2020 | Development and validation of clinical outcome assessments in LGMD2A1R21TR003184-01 · under PAR-18-953 | JOHNSON, NICHOLAS ELWOODSTATLAND, JEFFREY | VIRGINIA COMMONWEALTH UNIVERSITY, VA | $254,174 | ZTR1 |
| 2020 | Magnetic resonance and ultrasound imaging as biomarkers for detection and monitoring of Parsonage-Turner Syndrome (PTS)1R21TR003033-01A1 · under PAR-18-953 | SNEAG, DARRYL | HOSPITAL FOR SPECIAL SURGERY, NY | $251,421 | ZTR1 |
| 2020 | Methods to Improve Clinical Trials for A-T1R21TR003534-01 · under PAR-18-953 | LEDERMAN, HOWARD M.LEFTON-GREIF, MAUREEN ANATALE, VALERIE | JOHNS HOPKINS UNIVERSITY, MD | $244,900 | ZTR1 |
| 2020 | Proof of concept treatment interventions in a rodent model of the rare disease bronchiolitis obliterans1R21TR003037-01A1 · under PAR-18-953 | PALMER, SCOTT M | DUKE UNIVERSITY, NC | $241,500 | ZTR1 |
| 2020 | Facilitating personalized medicine of monogenic stone patients by genetic characterization1R21TR003174-01 · under PAR-18-953 | HARRIS, PETER C.LIESKE, JOHN C | MAYO CLINIC ROCHESTER, MN | $238,500 | ZTR1 |
| 2020 | Preclinical evaluation of the efficacy and mechanism of action for an alkylated polyamine analogue diethylnorspermine in treating pheochromocytoma/paraganglioma1R21TR003044-01A1 · under PAR-18-953 | GHAYEE, HANS K. | UNIVERSITY OF FLORIDA, FL | $228,750 | ZTR1 |
| 2020 | Tumor Immune Profiling to Optimize Clinical Trial Readiness in Basal Cell Nevus Syndrome1R21TR003341-01 · under PAR-18-953 | BICKERS, DAVID RINSEY | COLUMBIA UNIVERSITY HEALTH SCIENCES, NY | $202,500 | ZTR1 |
| 2020 | Clinical Trial Readiness for K channel inhibitors in Cantu Syndrome1R21HD103347-01 · under PAR-18-953 | GRANGE, DOROTHY KATHERINENICHOLS, COLIN G | WASHINGTON UNIVERSITY, MO | $196,875 | ZTR1 |
| 2019 | Biomarkers to enable ASO prevention trials in genetic prion disease carriers1R21TR003040-01 · under PAR-18-953 | ARNOLD, STEVEN E | MASSACHUSETTS GENERAL HOSPITAL, MA | $271,761 | ZTR1 |
| 2019 | Frataxin measurement for clinical trial readiness in Friedreich ataxia1R21TR003035-01 · under PAR-18-953 | LYNCH, DAVID ROBINSON | CHILDREN'S HOSP OF PHILADELPHIA, PA | $267,601 | ZTR1 |
| 2019 | Systemic biomarkers of brain injury from hyperammonemia1R21TR003166-01 · under PAR-18-953 | AH MEW, NICHOLASCALDOVIC, LJUBICA MORIZONO | CHILDREN'S RESEARCH INSTITUTE, DC | $260,407 | ZTR1 |
| 2019 | Distensibility and Remodeling as markers of Therapeutic Response in Eosinophilic Esophagitis1R21TR003039-01 · under PAR-18-953 | MENARD-KATCHER, CALIES DMUIR, AMANDA BROOKE | CHILDREN'S HOSP OF PHILADELPHIA, PA | $242,750 | ZTR1 |
| 2019 | Evaluating the psychometric properties of three clinical trial outcome measures for Rett syndrome1R21HD101075-01 · under PAR-18-953 | BYIERS, BREANNE J | UNIVERSITY OF MINNESOTA, MN | $231,288 | ZTR1 |
| 2019 | Clinical Trial Readiness for Rare Diseases, Disorders and Syndromes1R21TR003034-01 · under PAR-18-953 | SHY, MICHAEL E. | UNIVERSITY OF IOWA, IA | $209,000 | ZTR1 |
Data: Grants.gov's daily extract and NIH's notice pages and award files, in ScienceGraph release conductgrants-2026-11-09-0.1.0. An award is listed only where NIH's own record names the notice, and a PI only by NIH's profile ID. Grants.gov and NIH do not endorse ScienceDex.