Photo of David A. Weinstein, M.D., M.Sc.

David A. Weinstein, M.D., M.Sc.

Professor of Medicine
Academic Office Location:
UConn Health
263 Farmington Avenue
Farmington, CT 06030
Phone: 860-837-7800
B.S.Trinity CollegeBiology
M.D.Harvard Medical SchoolMedicine
M.Sc.Massachusetts Institute of Technology/Harvard Medical SchoolClinical Investigation

Post-Graduate Training
InternshipChildren's Hospital BostonPediatrics
ResidencyChildren's Hospital BostonPediatrics (Chief Resident, 1997-1998)
FellowshipChildren's Hospital BostonEndrocrinology
Clinical InvestigatorMIT/HarvardClinical Investigator Training Program

Name of Award/HonorAwarding Organization
Best Doctors in Connecticut (2018 - 2020)Connecticut Magazine
Named in "Best Doctors in America" 2007 - 2020
Chairman's Award for InnovationConnecticut Children's
"Be My Sugar" Research AwardCongenital Hyperinsulinism International
Presidencia de Honor AwardGlucolatino, España
Clinical Research AwardUniversity of Florida College of Medicine
Lifetime Service AwardAssociation for Glycogen Storage Disease
Award for Dedication to the GSD Community (2014-2015)Glucolatino Columbia
Amigo de las Glucogenosis AwardAsociación Española de Enfermos de Glucogenosis
Inducted into Rare Disease Research Hall of Fame
Recipient of Order of the Smile Humanitarian Award (for Efforts to Help Children from Around the World)Order Usmiechu in Poland
Jan Albrecht AwardAmerican Association for the Study of Liver Diseases
Padrino de HonorFundacaion Glucolatino
George A. Sacher Award for Geriatric ResearchGerontological Society of America
Trinity College Alumni Award (Recognizing 5 Graduates for Excellence in Scholastic, Athletic, and Service Endeavors)Trinity College
Barry M. Goldwater Scholarship Recipient for the State of Florida (Congressional Award Granted Annually to One Student per State Who Demonstrates “Outstanding Potential in the Fields of Mathematics and the Natural Sciences”)
Holland ScholarTrinity College

Dr. Weinstein specializes in disorders of carbohydrate metabolism with particular focus on glycogen storage disease (GSD), hypoglycemia, and diabetes. As the director of the Glycogen Storage Disease Program, he manages the inpatient and outpatient care for the largest cohort of GSD patients in the world following over 500 people with this disorder. Of these, over 225 patients have glycogen storage disease type Ia, 70 patients have glycogen storage disease type Ib, 50 patients have type III glycogen storage disease, and over 200 patients with types 0, VI, or IX GSD. His patients come from over 49 states and 43 countries to be cared for by him. Dr. Weinstein attends for the majority of glycogen storage disease inpatient evaluations along with 1-2 months per year on the inpatient endocrinology service.

Name & DescriptionCategoryRoleTypeScopeStart YearEnd Year
Clinical Advisory Committee, mRNA Trial (UT-053), UltragenyxResearch CommitteeExternalInternational2020
American Society of Gene and Cell TherapyProfessional/Scientific OrganizationNational2019
Scientic Advisory Committee, Ketotic Hypoglycemia InternationalAdvisory CommitteeInternational2019
Scientific Organizing Committee, 2019 International GSD Conference, Porto Alegre, Brazil.Education CommitteeExternalInternational20182019
James Lind Alliance Priority Setting Partnership Steering Committee (United Kingdom)Professional/Scientific OrganizationExternalInternational20172019
NICHD Pediatrics (CHHD-A) Review Committee (Permanent Member)Study SectionExternalNational2016
Clinical Advisory Committee, DTX401 Trial, Ultragenyx TherapeuticsResearch CommitteeChairmanExternalInternational2015
Scientific Advisory Committee, Dimension Therapeutics / Ultragenyx TherapeuticsAdvisory CommitteeExternalInternational2015
DSMB Member, New Drug Trial, PfizerAdvisory CommitteeMemberExternalNational20052009
Association for Glycogen Storage DiseaseAdvisory CommitteeBoard of DirectorsExternalNational20022007
Association for Glycogen Storage Disease (United Kingdom)Professional/Scientific OrganizationExternalInternational2002
Association for Glycogen Storage Disease (United States)Professional/Scientific OrganizationExternalNational1999
GLYDE Trial Steering Committee, Vitaflo CorporationResearch CommitteeVice ChairExternalInternational
Rare Disease Task Force for the State of ConnecticutAdvisory CommitteeExternalState
Scandinavian Association for Glycogen Storage DiseaseProfessional/Scientific OrganizationExternalInternational

Understanding the pathophysiology of hypoglycemia in children with a focus on the causes of ketotic hypoglycemia.

Investigation into the cause of morbidity type 0 and I glycogen storage disease including the pathophysiology of hepatic adenomas, hepatocellular carcinoma, nephrolithiasis, anemia, and focal segmental glomerulosclerosis.

Attempted methods of repairing the enzymatic defect in the glycogen storage diseases.

Journal Articles


  • Response letter.
    Lee, Youngmok; Chou, Janice Y; Weinstein, David A Journal of inherited metabolic disease 2018 Sep;41915


Title or AbstractTypeSponsor/EventDate/YearLocation
Gene therapy for glycogen storage disease type Ia: returning to Cali with progress (Feb 17, 2020)LectureIII Congresos de Glucogenosis Hepaticas en Colombia / Fuglucol Fundacion2020Cali, Colombia
AAV8-mediated liver-directed gene therapy as a potential therapeutic option in adults with glycogen storage disease type Ia (GSDIa): Results from a phase 1/2 clinical trial (May 15, 2020)Plenary LectureAmerican Society of Gene and Cell Therapy (ASGCT)2020Boston (virtual)
Glycogen storage disease type Ia: 30 years of progress from cornstarch to gene therapyLectureBoston Children's Hospital2020Boston, MA
Gene Therapy for Glycogen Storage Disease: Delivering on a PromiseLectureGlucolatino Ecuador2019Quito, Ecuador
Research Update: Gene Therapy and Other Progress since 2015LectureFuglucol and Glucolatino2019Cali, Colombia
Positive Cohort 1 and 2 Results from the Phase 1/2 Trial with AAV-8 Mediated Liver-Directed Gene Therapy in Adults with Glycogen Storage Disease Type IaPlenary Lecture5th International Glycogen Storage Diseases Conference2019Porto Alegre, Brazil
Returning to Argentina with Progress: Gene Therapy and a Research UpdateLectureGlucolatino2019Rosario, Argentina
Gene Therapy for Glucogenosis Typo Ia: Returning to Rosario with ProgressLectureFacultad de Ciencias Medicas2019Rosario, Argentina
Glycogen Storage Disease: Evolution of Treatment from Continuous Feeds to Gene TherapyLectureAssociatia Persoanelor cu Glicogenoza Conference2019Cluj-Napoca, Romania
Returning to Rimini with Progress: A Gene Therapy UpdateLectureAssociazione Italiana Glicogenosi2019Rimini, Italy
Research Update for the Ketotic Forms of GSD: Evolving TherapiesLectureAsociacion SHG Glycogenose2019Duderstadt, Germany
Gene Therapy for Glycogen Storage Disease Type Ia: Update 10 Months After the Initial TreatmentLectureAsociacion SHG Glycogenose2019Duderstadt, Germany
Update on the DTX401 Phase I/II Gene Therapy TrialLectureAssociation for Glycogen Storage Disease2019Houston, Texas
Gene Therapy Update: Culmination of 20 Years of ProgressLectureFourteenth Annual Children's Fund For GSD Fund-Raiser2019Fort Lauderdale, Florida
Glycogen Storage Disease: 30 Years of Progress from Cornstarch to Gene TherapyLectureGenetics/Metab Grand Rounds, Baylor Univ School of Medicine2019Houston, Texas
Ketotic Hypoglycemia: Re-evaluation of a Classic Pediatric ConditionLectureSecond Annual Joint Pediatric Symposium2019Danbury Hospital, Danbury, CT
The GLYDE Trial: A Comparison of Glycosade and uncooked cornstarch for the treatment of hepatic glycogen storage diseaseOtherSociety for the Study of Inborn Errors of Metabolism2019Rotterdam, Netherlands
An Open Label Phase 1/2 Clinical Trial for AAV8-Mediated Liver-Directed Gene Therapy in Adults with Glycogen Storage Disease Type IaPosterSociety for Pediatric Research2019Baltimore, Maryland
Positive Cohort 1 Results from the Phase 1/2 Trial with AAV8-Mediated Liver Directed Gene Therapy in Adults with Glycogen Storage Disease Type IaPlenary LectureAmerican Society for Gene and Cell Therapies2019Washington, DC
Metabolic Needs of Adult Patients with Glycogen Storage Disease Type IaPosterSociety for Pediatric Research2019Baltimore, Maryland
Biochemical and Temporal Profiling of Chemotherapy Associated HypoglycemiaPosterSociety for Pediatric Research2019Baltimore, Maryland
Effects of PhK Deficiency on Mouse Model of Glycogen Storage Disease Type IXPosterAmerican Society of Gene and Cell Threapy2019Washington, DC
Positive Cohorts 1 & 2 Results from the Phase 1/2 AAV8-Mediated Liver-Directed Gene Therapy TrialPlenary LectureSociety for the Study of Inborn Errors of Metabolism2019Rotterdam, The Netherlands
Non-Ketotic Glycogen Storage Disease UpdatePlenary Lecture5th International Glycogen Storage Diseases Conference2019Porto Alegre, Brazil
Glycogen storage disease clinical update: Progress from cornstarch to gene therapyLectureNew England Regional Genetics Group2019Portsmouth, New Hampshire
Returning to Malaga with Progress: A Gene Therapy UpdateLectureX Congreso Internacional de Glucogenosis2019Benalmadena, Spain
DTX 401 Phase I/II Gene Therapy Study: Lessons Learned.LectureUltragenyx European Investigator Meeting2018Madrid, Spain
Gene therapy for glycogen storage disease type IaLectureAssociation for Glycogen Storage Disease (UK)2018Wyboston Lakes, United Kingdom
Research update for types III, IV, VI, and IX glycogen storage disease.LectureIX Congreso Iberoamericano de Glucogenosis.2018Fuengirola, Spain
Gene therapy for glycogen storage disease type Ia.LectureIX Congreso Iberoamericano de Glucogenosis.2018Fuengirola, Spain
Forty Years of Progress: Reflections on Returning to IowaLectureAssociation for Glycogen Storage Disease Annual Conference2018Davenport, IA
Glycogen storage disease type I: From glycogen to gene therapy (and everything in-between),LectureGenetic Metabolic Dietitians International2018Orlando, FL
Hepatic glycogen storage: Prevention of Complications and Future Therapies.LectureScandinavian Association for Glycogen Storage Disease.2018Angelholm, Sweden
Research Update: Gene Therapy and MoreLectureAsociación SHG Glycogenose.2018Duderstadt, Germany
Born This Way, Hepatic Glycogen Storage Disease UpdateLectureVIA Symposium2018Berlin, Germany
Reflections on Returning to Trinity: Lessons from a Rare Disease.LectureTrinity College / Health Sciences Student Seminar Series2018Hartford, CT