The notices it replaced
- Reissues: PAR-22-101 Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
- 2 steps back: PAR-18-953 Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Replaced by PAR-25-450.
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 | 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 | 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 | SEVAGAMOORTHY, ANJANA | CHILDREN'S HOSP OF PHILADELPHIA, PA | $415,245 | ZTR1 |
| 2025 | Trial readiness for LCAT deficiency1R21TR005433-01 | CUCHEL, MARINA | UNIVERSITY OF PENNSYLVANIA, PA | $401,566 | ZTR1 |
| 2025 | 4-hydroxymandelate for HPDL deficiency, a novel mitochondrial encephalopathy1R21TR006111-01A1 | 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 | 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 | COLES, THERESAONWUEMENE, OLUWATOYOSI ADEFUNKE | DUKE UNIVERSITY, NC | $456,348 | ZTR1 |
| 2024 | Triangulating outcome assessments for blepharospasm1R21TR005231-01 | PETERSON, DAVID A | UNIVERSITY OF CALIFORNIA, SAN DIEGO, CA | $450,836 | ZTR1 |
| 2024 | Clinical Trial Readiness for Posterior Cortical Atrophy Syndrome1R21TR004695-01A1 | PELAK, VICTORIA SUSAN | UNIVERSITY OF COLORADO DENVER, CO | $429,000 | ZTR1 |
| 2024 | AI/ML and iPSC-Derived Organoids for Myotonic Dystrophy Drug Discovery1R21TR004938-01 | 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.