|
08-2030
|
MR Assessment of the effect of nNOS on Bioenergetics and Microvascular Function in Becker and Duchenne Muscular Dystrophy
MR Assessment of the effect of nNOS on Bioenergetics and Microvascular Function in Becker and Duchenne Muscular Dystrophy
|
|
07-2030
|
Addressing Shortcomings of AAV GeneTherapies for the Muscular Dystrophies
Addressing Shortcomings of AAV GeneTherapies for the Muscular Dystrophies
|
|
08-2029
|
Early Cardiac Biomarkers of Inflammation and Remodeling in DMD
Early Cardiac Biomarkers of Inflammation and Remodeling in DMD
|
|
05-2028
|
Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs
Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs
|
|
10-2027
|
MRI/MRS Markers of Skeletal Muscle Disease Progression and Vascular Function in Non-Ambulatory Individuals with DMD
MRI/MRS Markers of Skeletal Muscle Disease Progression and Vascular Function in Non-Ambulatory Individuals with DMD
|
|
06-2027
|
The MDA Care Center at University of Florida Health: MDA Care Center Grant Recertification
The MDA Care Center at University of Florida Health: MDA Care Center Grant Recertification
|
|
02-2027
|
PC8.1-135: Integrating High-Throughput Chromatography and Data Science in a Platform for the Rapid and Accurate Quantification of Full, Partial, and Empty AAV Capsids
PC8.1-135: Integrating High-Throughput Chromatography and Data Science in a Platform for the Rapid and Accurate Quantification of Full, Partial, and Empty AAV Capsids
|
|
01-2027
|
Development and optimization of a gene therapy vector for MEPAN Syndrome
Development and optimization of a gene therapy vector for MEPAN Syndrome
|
|
08-2026
|
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich#s Ataxia
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich#s Ataxia
|
|
08-2026
|
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
|
08-2026
|
Development of a Novel AAV Vector Capsid Optimized for OA Gene Therapy
Development of a Novel AAV Vector Capsid Optimized for OA Gene Therapy
|
|
06-2026
|
Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type B
Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type B
|
|
06-2026
|
UF Health Center for Advanced Therapeutics
UF Health Center for Advanced Therapeutics
|
|
06-2026
|
CMS Newborn Screening - COQWX
CMS Newborn Screening - COQWX
|
|
05-2026
|
Novel Gene Therapy for Critical Limb Ischemia and Limb Salvage in Vascular Occlusive Disease
Novel Gene Therapy for Critical Limb Ischemia and Limb Salvage in Vascular Occlusive Disease
|
|
04-2026
|
Frataxin Potency assay in support of an AAV-mediated gene therapy treatment for Friedreich#s ataxia
Frataxin Potency assay in support of an AAV-mediated gene therapy treatment for Friedreich#s ataxia
|
|
04-2026
|
Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa-fcab in Boys with DMD Prior to and Following AAV Gene Therapy
Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa-fcab in Boys with DMD Prior to and Following AAV Gene Therapy
|
|
04-2026
|
Control of Breathing and Pompe Disease
Control of Breathing and Pompe Disease
|
|
03-2026
|
Vector Biodistribution in Feline Tissue
Vector Biodistribution in Feline Tissue
|
|
01-2026
|
Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa- fcab in Boys with DMD Prior to and Following AAV Gene Therapy (MDA 1020019)
Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa- fcab in Boys with DMD Prior to and Following AAV Gene Therapy (MDA 1020019)
|
|
01-2026
|
Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa- fcab in Boys with DMD Prior to and Following AAV Gene Therapy (MDA 1020019)
Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa- fcab in Boys with DMD Prior to and Following AAV Gene Therapy (MDA 1020019)
|
|
01-2026
|
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich#s Ataxia
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich#s Ataxia
|
|
12-2025
|
MDA MOVR Data Hub Participation Agreement
MDA MOVR Data Hub Participation Agreement
|
|
12-2024
|
Task Order#1 for UF #AGR00019321- Rocket Pharmaceuticals
Task Order#1 for UF #AGR00019321- Rocket Pharmaceuticals
|
|
06-2024
|
MDA Care Center
MDA Care Center
|
|
03-2024
|
Preclinical sub-chronic toxicology and biodistribution study of adeno-associated virus (scAAV9-CBh-hAGAopt) mediated gene therapy for Aspartylglucosaminuria Disease in Sprague Dawley rats
Preclinical sub-chronic toxicology and biodistribution study of adeno-associated virus (scAAV9-CBh-hAGAopt) mediated gene therapy for Aspartylglucosaminuria Disease in Sprague Dawley rats
|
|
11-2023
|
Optimizing AAV Vectors for Central Nervous System transduction
Optimizing AAV Vectors for Central Nervous System transduction
|
|
08-2023
|
Task Order #10 - Construct Design and Generation Collaboration
Task Order #10 - Construct Design and Generation Collaboration
|
|
07-2023
|
Cincinnati Zoo Feline and Canine Shedding 2022 Non-GLP
Cincinnati Zoo Feline and Canine Shedding 2022 Non-GLP
|
|
06-2023
|
SRA - Project Planning
SRA - Project Planning
|
|
06-2023
|
An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
|
|
05-2023
|
Immune response to AAV Vector Capsid and Assessment of Eligibility for AAV-mediated Gene Therapy for Duchenne Muscular Dystrophy
Immune response to AAV Vector Capsid and Assessment of Eligibility for AAV-mediated Gene Therapy for Duchenne Muscular Dystrophy
|
|
04-2023
|
Support of NIH catalyze Award for AAV manufacturing and GLP Toxicology studies
Support of NIH catalyze Award for AAV manufacturing and GLP Toxicology studies
|
|
02-2023
|
Task Order # 12 Pompe Disease Research Support
Task Order # 12 Pompe Disease Research Support
|
|
12-2022
|
Sarepta TO #1: Support of PGTC Cores
Sarepta TO #1: Support of PGTC Cores
|
|
11-2022
|
Development of a novel gene therapy for lupus
Development of a novel gene therapy for lupus
|
|
07-2022
|
Breathing Research and Therapeutics (BREATHE)
Breathing Research and Therapeutics (BREATHE)
|
|
06-2022
|
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research - F017885/6
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research - F017885/6
|
|
06-2022
|
Cure FA Research Foundation
Cure FA Research Foundation
|
|
05-2022
|
An open-label, multicenter, multinational, extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
An open-label, multicenter, multinational, extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
|
|
04-2022
|
A Long-term Study to Evaluate Growth and Development Outcomes in Patients with Infantile-Onset Pompe Disease Who Are Receiving Myzoyme (alglucosidase alfa)
A Long-term Study to Evaluate Growth and Development Outcomes in Patients with Infantile-Onset Pompe Disease Who Are Receiving Myzoyme (alglucosidase alfa)
|
|
03-2022
|
Expanded access trial of systemic delivery of aspartoacylase (rAAV0-CB6-AspA) gene vector in a single patient with canavan disease
Expanded access trial of systemic delivery of aspartoacylase (rAAV0-CB6-AspA) gene vector in a single patient with canavan disease
|
|
12-2021
|
TO #1 - Access and Transfer of rAAV9-CMV-hAADC Production and Testing Methods#
TO #1 - Access and Transfer of rAAV9-CMV-hAADC Production and Testing Methods#
|
|
08-2021
|
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy
|
|
07-2021
|
Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease
Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease
|
|
07-2021
|
Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat
Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat
|
|
07-2021
|
Task Order #4 - Assay Development Support for Friedreich#s Ataxia
Task Order #4 - Assay Development Support for Friedreich#s Ataxia
|
|
05-2021
|
Determination of anti-AAV9 IgG & anti-AAV9 IgM level in mouse serum samples
Determination of anti-AAV9 IgG & anti-AAV9 IgM level in mouse serum samples
|
|
05-2021
|
Data Registry Services
Data Registry Services
|
|
03-2021
|
GLP Tissue and Shedding Biodistribution Study in Nonhuman Primates
GLP Tissue and Shedding Biodistribution Study in Nonhuman Primates
|
|
12-2020
|
Analysis of Cincinnati Zoo Feline Shedding
Analysis of Cincinnati Zoo Feline Shedding
|
|
12-2020
|
Non-GLP Expression Study in C57BL/6 Mice
Non-GLP Expression Study in C57BL/6 Mice
|
|
12-2020
|
Analysis of Human Samples for Biodistribution and Circulating Antibodies
Analysis of Human Samples for Biodistribution and Circulating Antibodies
|
|
12-2020
|
Sarepta Task Order #7 - Pompe Canine Colony Housing Program
Sarepta Task Order #7 - Pompe Canine Colony Housing Program
|
|
12-2020
|
Fox Research Fund
Fox Research Fund
|
|
12-2020
|
Sunshine Children's Fund
Sunshine Children's Fund
|
|
12-2020
|
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research -
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research -
|
|
09-2020
|
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
|
08-2020
|
UFRCC for Cardiovascular Cell Therapy Research Network
UFRCC for Cardiovascular Cell Therapy Research Network
|
|
06-2020
|
Solid TO #15 Evaluation of Immune Management in AAV-?Dys Gene Therapy
Solid TO #15 Evaluation of Immune Management in AAV-?Dys Gene Therapy
|
|
04-2020
|
Antisense oligonucleotide therapy for a novel humanized GAA rat model of Pompe Disease
Antisense oligonucleotide therapy for a novel humanized GAA rat model of Pompe Disease
|
|
12-2019
|
Task Order #13 - Evaluation of SGT-01 in Neonatal NHP using Immune Modulation
Task Order #13 - Evaluation of SGT-01 in Neonatal NHP using Immune Modulation
|
|
12-2019
|
GLP Tissue-only Biodistribution Study in Nonhuman Primates
GLP Tissue-only Biodistribution Study in Nonhuman Primates
|
|
12-2019
|
Solid TO#12 - Study ID:UFGTC15-007/ UFGTC16-007/UFGTC16-008
Solid TO#12 - Study ID:UFGTC15-007/ UFGTC16-007/UFGTC16-008
|
|
12-2019
|
Neuromuscular Disease/Clinical Trial Support Fund
Neuromuscular Disease/Clinical Trial Support Fund
|
|
11-2019
|
Process Development for AAV Manufacturing of AAV-CB-hFXN
Process Development for AAV Manufacturing of AAV-CB-hFXN
|
|
06-2019
|
Respiratory Muscle Function in Untreated X-linked Myotubular Myopathy
Respiratory Muscle Function in Untreated X-linked Myotubular Myopathy
|
|
03-2019
|
Toxicology Study of BS01 in Rd1 mice
Toxicology Study of BS01 in Rd1 mice
|
|
03-2019
|
OneFlorida Clinical Research Consortium Phase II
OneFlorida Clinical Research Consortium Phase II
|
|
03-2019
|
POM002 LAB SCERVICES AGREEMENT - GAA ACTIVITY ASSAY ANDGLYCOGEN BIOCHEMICAL ASSAY TESTS
POM002 LAB SCERVICES AGREEMENT - GAA ACTIVITY ASSAY ANDGLYCOGEN BIOCHEMICAL ASSAY TESTS
|
|
01-2019
|
Diaphragm pacing and rehabilitation in Pompe disease
Diaphragm pacing and rehabilitation in Pompe disease
|
|
12-2018
|
Lacerta Task Order #3 - NHP TAb Screening
Lacerta Task Order #3 - NHP TAb Screening
|
|
12-2018
|
Limelight TO #1 - Optimizing Hydrodynamic Transduction Injection Procedure
Limelight TO #1 - Optimizing Hydrodynamic Transduction Injection Procedure
|
|
12-2018
|
Analysis of Cincinnati Zoo Feline Shedding
Analysis of Cincinnati Zoo Feline Shedding
|
|
12-2018
|
BSF Human Material Storage
BSF Human Material Storage
|
|
10-2018
|
McGollie - Longitudinal, Observations Study of Subjects with Cardioskeletal Myopathy
McGollie - Longitudinal, Observations Study of Subjects with Cardioskeletal Myopathy
|
|
08-2018
|
The relationship between genomic variants and MRI/MRS markers in DMD
The relationship between genomic variants and MRI/MRS markers in DMD
|
|
08-2018
|
SOLID TO #5 ASSESSMENT OF SAFETY/EFFICACY OF IVAAV-MICRO-DYSTROPHIN CONSTRUCT
SOLID TO #5 ASSESSMENT OF SAFETY/EFFICACY OF IVAAV-MICRO-DYSTROPHIN CONSTRUCT
|
|
08-2018
|
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
|
|
07-2018
|
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
|
|
06-2018
|
Hex-003-004
Hex-003-004
|
|
06-2018
|
Development of a Strength Training Protocol in Duchenne Muscular Dystrophy
Development of a Strength Training Protocol in Duchenne Muscular Dystrophy
|
|
06-2018
|
Control of Breathing and Pompe Disease
Control of Breathing and Pompe Disease
|
|
05-2018
|
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-US-201
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-US-201
|
|
04-2018
|
A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease
A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease
|
|
02-2018
|
A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale
A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale
|
|
02-2018
|
Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy
Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy
|
|
12-2017
|
A Medical Chart Review of Patients with X-Linked Myotubular Myopathy (XLMTM)
A Medical Chart Review of Patients with X-Linked Myotubular Myopathy (XLMTM)
|
|
09-2017
|
MD110050 Optical Imaging of Dystrophic and Damaged Muscle
MD110050 Optical Imaging of Dystrophic and Damaged Muscle
|
|
07-2017
|
Production and Testing of rAAV9-DES-hGAA Clinical Product and rAAV9 Excipient TO#2
Production and Testing of rAAV9-DES-hGAA Clinical Product and rAAV9 Excipient TO#2
|
|
06-2017
|
Solid Fellow
Solid Fellow
|
|
06-2017
|
A Pilot Study of the Effects of Oral Administration of Zavesca on Anti-rhGAA Immune Response in Subjects with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
A Pilot Study of the Effects of Oral Administration of Zavesca on Anti-rhGAA Immune Response in Subjects with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
|
|
05-2017
|
Heart and Skeletal Muscle Metabolism, Energetics and Function in Barth Syndrome
Heart and Skeletal Muscle Metabolism, Energetics and Function in Barth Syndrome
|
|
04-2017
|
A Long Term Study for Extended BMN 701 Treatement of Patients with Pompe Disease who Have Particpated in a BMN701 Study
A Long Term Study for Extended BMN 701 Treatement of Patients with Pompe Disease who Have Particpated in a BMN701 Study
|
|
03-2017
|
Development therapeutic gene therapy vector for N-glycanase 1-Deficiency
Development therapeutic gene therapy vector for N-glycanase 1-Deficiency
|
|
02-2017
|
Response to Diaphragmatic Pacing in Subjects with Pompe
Response to Diaphragmatic Pacing in Subjects with Pompe
|
|
12-2016
|
Project Development Planning
Project Development Planning
|
|
12-2016
|
Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy in Rat Model of Pompe Disease # Immunomodulation Study
Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy in Rat Model of Pompe Disease # Immunomodulation Study
|
|
12-2016
|
AAV9 Pompe Rodent Study
AAV9 Pompe Rodent Study
|
|
11-2016
|
A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) in rhGAA Exposed Subjects with Late-onset Pompe Disease
A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) in rhGAA Exposed Subjects with Late-onset Pompe Disease
|
|
08-2016
|
Clinical and Translational Science Institute
Clinical and Translational Science Institute
|
|
08-2016
|
A RANDOMED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 TRIAL OF TADALAFIL FOR DUCHENNE MUSCULAR DYSTROPHY
A RANDOMED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 TRIAL OF TADALAFIL FOR DUCHENNE MUSCULAR DYSTROPHY
|
|
06-2016
|
Development of Clinical Translational Studies
Development of Clinical Translational Studies
|
|
05-2016
|
Request for Support to Plan Clinical Gene Therapy Program for BTHS
Request for Support to Plan Clinical Gene Therapy Program for BTHS
|
|
03-2016
|
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
|
|
03-2016
|
Imaging Core: Protocol 701-301
Imaging Core: Protocol 701-301
|
|
03-2016
|
MP3 Conference Fund
MP3 Conference Fund
|
|
02-2016
|
Pompe Gene Therapy
Pompe Gene Therapy
|
|
01-2016
|
AGLU07510 PEAS
AGLU07510 PEAS
|
|
09-2015
|
A Phase III Double-Blind, Randomised, Placebo-Controlled
A Phase III Double-Blind, Randomised, Placebo-Controlled
|
|
08-2015
|
Assessment of Cardiopulmonary Function in Duchenne Muscu
Assessment of Cardiopulmonary Function in Duchenne Muscu
|
|
08-2015
|
Magnetic Resonance Imaging and Biomarkers for Muscular D
Magnetic Resonance Imaging and Biomarkers for Muscular D
|
|
07-2015
|
Failed Regeneration in the Muscular Dystrophies: Inflamm
Failed Regeneration in the Muscular Dystrophies: Inflamm
|
|
05-2015
|
Post-doctoral training in glycogen storage disease type
Post-doctoral training in glycogen storage disease type
|
|
05-2015
|
NEOGAA Clinical Trial
NEOGAA Clinical Trial
|
|
02-2015
|
Pompe Research - UF Matching Account
Pompe Research - UF Matching Account
|
|
01-2015
|
Treatment of Pompe Disease via Retrograde Transduction o
Treatment of Pompe Disease via Retrograde Transduction o
|
|
11-2014
|
AT2220-010: An Open-Label, Multi-Center, International
AT2220-010: An Open-Label, Multi-Center, International
|
|
10-2014
|
Amicus Data Set Agrmt
Amicus Data Set Agrmt
|
|
09-2014
|
Upper airway gene therapy in a mouse model of Pompe dise
Upper airway gene therapy in a mouse model of Pompe dise
|
|
08-2014
|
Spinal and brainstem respiratory neurons in Pompe diseas
Spinal and brainstem respiratory neurons in Pompe diseas
|
|
08-2014
|
In Vivo Characterization of Novel Modified Recombinant H
In Vivo Characterization of Novel Modified Recombinant H
|
|
07-2014
|
Failed Regeneration in the Muscular Dystrophies: Inflamm
Failed Regeneration in the Muscular Dystrophies: Inflamm
|
|
06-2014
|
Gene Therapy for Lung and Cardiovascular Disease
Gene Therapy for Lung and Cardiovascular Disease
|
|
05-2014
|
Planning a Multi-Center Clinical Trial of PDE5
Planning a Multi-Center Clinical Trial of PDE5
|
|
05-2014
|
A Phase 4 Prospective Exploratory Muscle Biopsy, Biomark
A Phase 4 Prospective Exploratory Muscle Biopsy, Biomark
|
|
12-2013
|
Oxyrane
Oxyrane
|
|
11-2013
|
A PHASE I OPEN-LABEL SAFETY/TOLERABILITY/ PK/PD STUDY OF
A PHASE I OPEN-LABEL SAFETY/TOLERABILITY/ PK/PD STUDY OF
|
|
09-2013
|
MRI Central Core Lab Services for POM001
MRI Central Core Lab Services for POM001
|
|
07-2013
|
Failed Regeneration in the Muscular Dystrophies
Failed Regeneration in the Muscular Dystrophies
|
|
06-2013
|
Phase I/II Trial of Diaphragm Delivery of aAAV1-CMV-GAA
Phase I/II Trial of Diaphragm Delivery of aAAV1-CMV-GAA
|
|
03-2013
|
Oral immune modulatory therapy using antigens bioencapsu
Oral immune modulatory therapy using antigens bioencapsu
|
|
01-2013
|
Exercise Video for Pompe patients
Exercise Video for Pompe patients
|
|
11-2012
|
Chatlos Gene Therapy Research - Companion Award
Chatlos Gene Therapy Research - Companion Award
|
|
11-2012
|
UFF Companion Award - Gene Therapy Research
UFF Companion Award - Gene Therapy Research
|
|
09-2012
|
Control of Breathing and Glycogen Storage
Control of Breathing and Glycogen Storage
|
|
09-2012
|
Gene Therapy for Cardiac and Skeletal Myopathies
Gene Therapy for Cardiac and Skeletal Myopathies
|
|
08-2012
|
MR Monitoring of PTC124 Treatment in DMD
MR Monitoring of PTC124 Treatment in DMD
|
|
07-2012
|
Failed Regeneration in the Muscular Dystrophies:
Failed Regeneration in the Muscular Dystrophies:
|
|
07-2012
|
CEDARS-SINAI MEDICAL CENTER CONSULTING SERVICES AGREEMEN
CEDARS-SINAI MEDICAL CENTER CONSULTING SERVICES AGREEMEN
|
|
06-2012
|
GENE THERAPY CENTER
GENE THERAPY CENTER
|
|
06-2012
|
Clinical Trial Support for Muscular Dystrophy Studies
Clinical Trial Support for Muscular Dystrophy Studies
|
|
06-2012
|
Alglucosidase ALFA Temporary Access Program
Alglucosidase ALFA Temporary Access Program
|
|
03-2012
|
GILT Tagged Enzyme Replacemetn Therapy for Pompe Disease
GILT Tagged Enzyme Replacemetn Therapy for Pompe Disease
|
|
02-2012
|
UFCC for Cardiovascular Cell Therapy Research Network
UFCC for Cardiovascular Cell Therapy Research Network
|
|
02-2012
|
GENE THERAPY IN MOUSE MODEL OF BARTH SYNDROME
GENE THERAPY IN MOUSE MODEL OF BARTH SYNDROME
|
|
02-2012
|
ENZYME ACTIVITY AND CRIM STATUS IN PATIENTS WITH LATE-ON
ENZYME ACTIVITY AND CRIM STATUS IN PATIENTS WITH LATE-ON
|
|
12-2011
|
Gene Therapy for Glycogen Storage Diseaes Type Ia Human
Gene Therapy for Glycogen Storage Diseaes Type Ia Human
|
|
10-2011
|
Failed Regeneration in the Muscular Dystrophies:
Failed Regeneration in the Muscular Dystrophies:
|
|
07-2011
|
Module I Clinical Trials of Gene Therapy for Leber Conge
Module I Clinical Trials of Gene Therapy for Leber Conge
|
|
07-2011
|
Recombinant AAV For Correction of Genetic Abnormalities
Recombinant AAV For Correction of Genetic Abnormalities
|
|
03-2011
|
Barth Syndrome Registry and DNA Bank
Barth Syndrome Registry and DNA Bank
|
|
10-2010
|
AAV-modified Human Skeletal Myoblast Therapy for Heart F
AAV-modified Human Skeletal Myoblast Therapy for Heart F
|
|
08-2010
|
Pilot Study to Determine the Efficacy of Newborn Screeni
Pilot Study to Determine the Efficacy of Newborn Screeni
|
|
07-2010
|
Clinical Trials of Gene Therapy for Leber Congenital Ama
Clinical Trials of Gene Therapy for Leber Congenital Ama
|
|
07-2010
|
Genzyme Support Services Agreement
Genzyme Support Services Agreement
|
|
06-2010
|
NGVL Toxicology Laboratory
NGVL Toxicology Laboratory
|
|
04-2010
|
Magnetic Resonance Imaging and BioMarkers for Muscular D
Magnetic Resonance Imaging and BioMarkers for Muscular D
|
|
04-2010
|
MRI Assessment of PTC124 Treatment in Boys with DMD
MRI Assessment of PTC124 Treatment in Boys with DMD
|
|
03-2010
|
An Open-Label, Multicenter Study of Oral AT2220 in Patie
An Open-Label, Multicenter Study of Oral AT2220 in Patie
|
|
03-2010
|
POM-CL-002 Multienter study to eval new method to measur
POM-CL-002 Multienter study to eval new method to measur
|
|
02-2010
|
Cardiac and Skeletal Muscle in Barth Syndrome: Evaluat
Cardiac and Skeletal Muscle in Barth Syndrome: Evaluat
|
|
08-2009
|
Preclinical Study of Adeno-Associated Virus Mediated Gen
Preclinical Study of Adeno-Associated Virus Mediated Gen
|
|
08-2009
|
NONINVASIVE MONITORING AND TRACKING OF MUSCLE STEM CELLS
NONINVASIVE MONITORING AND TRACKING OF MUSCLE STEM CELLS
|
|
07-2009
|
Clinical Trials of Gene Therqapy for Leber Congenital Am
Clinical Trials of Gene Therqapy for Leber Congenital Am
|
|
07-2009
|
Genzyme AGLU03206 Protocol
Genzyme AGLU03206 Protocol
|
|
05-2009
|
Pharmacological Chaperone Therapy for Pompe Disease
Pharmacological Chaperone Therapy for Pompe Disease
|
|
03-2009
|
GENE and PHARMACOLOGICAL THERAPIES FOR CYSTIC FIBROSIS PRO
GENE and PHARMACOLOGICAL THERAPIES FOR CYSTIC FIBROSIS PRO
|
|
08-2008
|
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
|
|
08-2008
|
Multicenter Study: Characterize the Effects of Pharmacol
Multicenter Study: Characterize the Effects of Pharmacol
|
|
07-2008
|
Clinical Trials of Gene Therqapy for Leber Congenital Am
Clinical Trials of Gene Therqapy for Leber Congenital Am
|
|
03-2008
|
Gene Therapy for GSD Type 1a
Gene Therapy for GSD Type 1a
|
|
12-2007
|
Gene therapy Treament of Pompe Disease with rAAV-GAA
Gene therapy Treament of Pompe Disease with rAAV-GAA
|
|
12-2007
|
Production of AAV1-AT
Production of AAV1-AT
|
|
08-2007
|
Clinical Trials of Gene Therapy for Leber Congential Ama
Clinical Trials of Gene Therapy for Leber Congential Ama
|
|
07-2007
|
Gene Therapy for Leber Congenital Amaurosis
Gene Therapy for Leber Congenital Amaurosis
|
|
06-2007
|
AAV1-AAT Part 1
AAV1-AAT Part 1
|
|
06-2007
|
AAV1-AAT Part 11
AAV1-AAT Part 11
|
|
07-2006
|
OPN-LBL MULTICTR STDY OF THE SAF EFFIC PHARMACOKINETEICS
OPN-LBL MULTICTR STDY OF THE SAF EFFIC PHARMACOKINETEICS
|
|
07-2006
|
AGLU02403 A Long-Term Continuation Study of Patients wi
AGLU02403 A Long-Term Continuation Study of Patients wi
|
|
06-2006
|
PHYSIOLOGICAL AND FUNCTIONAL ASSESSMENT OF RAAV-MEDICATE
PHYSIOLOGICAL AND FUNCTIONAL ASSESSMENT OF RAAV-MEDICATE
|
|
06-2006
|
INCREASED INOS AND REACTIVE OXYGEN SPECIES IN DIABETIC
INCREASED INOS AND REACTIVE OXYGEN SPECIES IN DIABETIC
|
|
04-2006
|
AGLU01602-AN OPEN-LABEL,MULTICENTER,MULTINATIONAL,DOSE-R
AGLU01602-AN OPEN-LABEL,MULTICENTER,MULTINATIONAL,DOSE-R
|
|
01-2006
|
Gene Therapy for GSD Type 1a
Gene Therapy for GSD Type 1a
|
|
09-2005
|
PRECLINICAL STUDY OF GENE REPLACEMENT THERAPY IN MUSCULA
PRECLINICAL STUDY OF GENE REPLACEMENT THERAPY IN MUSCULA
|
|
08-2005
|
NGVL Toxicology Laboratory
NGVL Toxicology Laboratory
|
|
08-2005
|
Phase I Study of Mini-Dystrophin Gene in AAV
Phase I Study of Mini-Dystrophin Gene in AAV
|
|
07-2005
|
Gene Therapy for Leber Congenital Amaurosis
Gene Therapy for Leber Congenital Amaurosis
|
|
07-2005
|
PROJ 3: TRANSDUCTION OF HEPATOCYTES WITH RECOMBINANT AAV
PROJ 3: TRANSDUCTION OF HEPATOCYTES WITH RECOMBINANT AAV
|
|
07-2005
|
Pfizer Visiting Professorship Program in Cardiovascular
Pfizer Visiting Professorship Program in Cardiovascular
|
|
02-2004
|
COMPARISON OF TWO RECOMBINANT ADENO-ASSOCIATED VIRUS VEC
COMPARISON OF TWO RECOMBINANT ADENO-ASSOCIATED VIRUS VEC
|
|
07-2003
|
GLYOCEN STORAGE DISEASE: HEPATIC GENE THERAPY
GLYOCEN STORAGE DISEASE: HEPATIC GENE THERAPY
|
|
06-2003
|
FUNCTIONAL ASSESSMENT OF ADEN0-ASSOCIATED VIRUS MEDIATED
FUNCTIONAL ASSESSMENT OF ADEN0-ASSOCIATED VIRUS MEDIATED
|
|
06-2003
|
INHIBITION OF HUMORAL IMMUNITY IN LIVER-TARGETED GENE
INHIBITION OF HUMORAL IMMUNITY IN LIVER-TARGETED GENE
|
|
03-2003
|
PRECLINICAL STUDIES OF ENZYME REPLACEMENT THERAPY FOR
PRECLINICAL STUDIES OF ENZYME REPLACEMENT THERAPY FOR
|
|
08-2002
|
PROJECT 2: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
PROJECT 2: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
|
|
08-2002
|
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
|
|
06-2002
|
GENE THERAPY APPLICATIONS FOR AN INHERITED CARDIOMYOPATH
GENE THERAPY APPLICATIONS FOR AN INHERITED CARDIOMYOPATH
|
|
06-2002
|
PREVENTION OF INHERITED CARDIOMYOPATHY IN A MURING MODEL
PREVENTION OF INHERITED CARDIOMYOPATHY IN A MURING MODEL
|
|
06-2002
|
PRECLINICAL STUDY FOR GENETIC CORRECTION OF INHERITED
PRECLINICAL STUDY FOR GENETIC CORRECTION OF INHERITED
|
|
06-2002
|
FUNCTIONAL ASSESSMANT OF ADENO-ASSOCIATED VIRUS MEDIATED
FUNCTIONAL ASSESSMANT OF ADENO-ASSOCIATED VIRUS MEDIATED
|
|
06-2002
|
EFFECTS OF INCREASED PROSTACYCLIN SYNTHASE EXPRESSION IN
EFFECTS OF INCREASED PROSTACYCLIN SYNTHASE EXPRESSION IN
|
|
06-2002
|
TREATMENT OF A MOUSE MODEL OF HEMOPHILIA A USING AAV VEC
TREATMENT OF A MOUSE MODEL OF HEMOPHILIA A USING AAV VEC
|
|
04-2002
|
PRECLINICAL STUDY OF PARVOVIRUS MEDIATED GENE THERAPY
PRECLINICAL STUDY OF PARVOVIRUS MEDIATED GENE THERAPY
|
|
01-2002
|
INDUSTION OF CARDIAC AND ENDITHELIAL ALLOGRAFT TOLERANCE
INDUSTION OF CARDIAC AND ENDITHELIAL ALLOGRAFT TOLERANCE
|
|
06-2001
|
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
|
|
03-2001
|
CARDIAC MUSCLE REGENERATION USING MESENCHYMAL STEM CELLS
CARDIAC MUSCLE REGENERATION USING MESENCHYMAL STEM CELLS
|
|
12-2000
|
CORRECTION OF INHERITED CARDIOMYOPATHY USING AAV VECTORS
CORRECTION OF INHERITED CARDIOMYOPATHY USING AAV VECTORS
|
|
11-2000
|
SCALEABLE PRODUCTION OF RAAV VECTORS
SCALEABLE PRODUCTION OF RAAV VECTORS
|
|
10-2000
|
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
|
|
09-2000
|
NEOCYTOLYSIS: MECHANISMS and LIMITATIONS
NEOCYTOLYSIS: MECHANISMS and LIMITATIONS
|
|
06-2000
|
CORRECTION OF INHERITED CARDIOMYOPATHY IN A MURINE EMBRY
CORRECTION OF INHERITED CARDIOMYOPATHY IN A MURINE EMBRY
|
|
06-2000
|
TREATMENT OF MOUSE MODEL OF HEMOPHILIA A USING AAV VECTO
TREATMENT OF MOUSE MODEL OF HEMOPHILIA A USING AAV VECTO
|
|
05-1999
|
GENE REPLACEMENT IN EMBRYONIC MODELS OF ACID MALTASE
GENE REPLACEMENT IN EMBRYONIC MODELS OF ACID MALTASE
|