Paul R. Harmatz
Elite in Mucopolysaccharidoses (MPS)

Dr. Paul R. Harmatz

Pediatric Gastroenterology | Gastroenterology
744 52nd St, 5th Floor, 
Oakland, CA 
Offers Telehealth
Elite in Mucopolysaccharidoses (MPS)
744 52nd St, 5th Floor, 
Oakland, CA 
OverviewInsuranceLocationsClinical Research

Overview

Dr. Paul Harmatz is a Pediatric Gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases. He is the Medical Director of the Pediatric Clinical Research Program in Mucopolysaccharidoses (MPS) and Related Disorders at UCSF Benioff Children's Hospital. Dr. Harmatz has been the Principal Investigator on multiple clinical trials involving MPS and its variants. He is licensed to practice in CA.

Dr. Harmatz is rated as an Elite provider by MediFind in the treatment of Mucopolysaccharidoses (MPS). His top areas of expertise are Mucopolysaccharidoses (MPS), Mucopolysaccharidosis Type 6 (MPS VI, Maroteaux-Lamy Syndrome), Mucopolysaccharidosis Type 2 (MPS II, Hunter Syndrome), and Mucopolysaccharidosis Type 4 (MPS IV, Morquio Syndrome).

His clinical research consists of co-authoring 169 peer reviewed articles and participating in 22 clinical trials. MediFind looks at clinical research from the past 15 years. In particular, he has co-authored 76 articles and participated in 5 clinical trials in the study of Mucopolysaccharidoses (MPS).

Residency
Los Angeles County-Harbor-UCLA Medical Center
Specialties
Pediatric Gastroenterology
Gastroenterology
Licenses
Pediatric Gastroenterology in CA
Board Certifications
American Board Of Pediatrics
Hospital Affiliations
UCSF Benioff Children's Hospital Oakland
Alta Bates Summit Medical Center
UCSF Medical Center
Languages Spoken
English
Gender
Male

Insurance

Accepted insurance can change. Please verify directly with the provider.

Find your insurance
Find your insuranceClose

Accepted insurance plans:

CareFirst
  • HMO
  • POS
  • PPO
Cigna
  • EPO
  • HMO
  • PPO
Humana
  • HMO
  • INDEMNITY
  • POS
  • PPO
Medicaid
  • OTHER MEDICAID
  • STATE MEDICAID
UnitedHealthcare
  • EPO
  • HMO
  • POS
  • PPO

Locations

UCSF Benioff Children's Hospital Gastroenterology Clinic
744 52nd St, 5th Floor, Oakland, CA 94609

Clinical Research

Clinical research consists of overseeing clinical studies of patients undergoing new treatments and therapies, and publishing articles in peer reviewed medical journals. Providers who actively participate in clinical research are generally at the forefront of the fields and aware of the most up-to-date advances in treatments for their patients.

22 Clinical Trials

A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants With Hunter Syndrome
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants With Hunter Syndrome
Enrollment Status: Active_not_recruiting
Publish Date: August 07, 2025
Intervention Type: Drug
Study Drug: DNL310
Study Phase: Phase 1/Phase 2
Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Enrollment Status: Active_not_recruiting
Publish Date: April 11, 2025
Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2
Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2
Enrollment Status: Completed
Publish Date: March 26, 2025
Intervention Type: Drug
Study Drug: Infigratinib
Study Phase: Phase 2
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Enrollment Status: Active_not_recruiting
Publish Date: January 28, 2025
Intervention Type: Genetic
Study Phase: Phase 2/Phase 3
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)
Enrollment Status: Completed
Publish Date: January 28, 2025
Intervention Type: Genetic
Study Phase: Phase 1/Phase 2
Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C
Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C
Enrollment Status: Completed
Publish Date: November 29, 2024
Intervention Type: Drug
Study Drug: Arimoclomol
Study Phase: Phase 2/Phase 3
A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
Enrollment Status: Completed
Publish Date: June 10, 2024
Intervention Type: Other
A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)
A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)
Enrollment Status: Completed
Publish Date: February 21, 2023
Intervention Type: Drug
Study Drug: AGT-181
Study Phase: Phase 1
A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)
A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)
Enrollment Status: Terminated
Publish Date: January 26, 2023
Intervention Type: Biological
Study Drug: SB-318
Study Phase: Phase 1/Phase 2
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Enrollment Status: Active_not_recruiting
Publish Date: October 26, 2022
A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)
A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)
Enrollment Status: Terminated
Publish Date: October 25, 2022
Intervention Type: Biological
Study Drug: SB-913
Study Phase: Phase 1/Phase 2
A Prospective, Observational Study of Pediatric Patients With Neuronopathic Forms of MPS II (Hunter Syndrome)
A Prospective, Observational Study of Pediatric Patients With Neuronopathic Forms of MPS II (Hunter Syndrome)
Enrollment Status: Withdrawn
Publish Date: October 10, 2022
Intervention Type: Other
A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase
A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase
Enrollment Status: Completed
Publish Date: June 10, 2021
Intervention Type: Biological
Study Phase: Phase 2/Phase 3
A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
Enrollment Status: Completed
Publish Date: June 10, 2021
Intervention Type: Biological
Study Phase: Phase 2/Phase 3
An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
Enrollment Status: Completed
Publish Date: June 10, 2021
Intervention Type: Biological
Study Phase: Phase 2/Phase 3
A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
Enrollment Status: Completed
Publish Date: June 08, 2021
Intervention Type: Biological
An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.
An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease.
Enrollment Status: No_longer_available
Publish Date: May 24, 2021
Intervention Type: Biological
MPS VI Clinical Surveillance Program (CSP)
MPS VI Clinical Surveillance Program (CSP)
Enrollment Status: Completed
Publish Date: April 05, 2021
A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
Enrollment Status: Completed
Publish Date: July 30, 2020
Intervention Type: Drug
Study Phase: Phase 3
Observational and Cross-Sectional Cohort Study of the Natural History and Phenotypic Spectrum of Farber Disease
Observational and Cross-Sectional Cohort Study of the Natural History and Phenotypic Spectrum of Farber Disease
Enrollment Status: Completed
Publish Date: January 18, 2020
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Enrollment Status: Completed
Publish Date: September 06, 2019
A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult Patients With Mucopolysaccharidosis II (MPS II, Hunter Syndrome)
A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult Patients With Mucopolysaccharidosis II (MPS II, Hunter Syndrome)
Enrollment Status: Completed
Publish Date: September 18, 2018
Intervention Type: Drug
Study Phase: Phase 1
View 21 Less Clinical Trials

169 Total Publications

Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Journal: Molecular genetics and metabolism
Published: March 17, 2025

Areas of Expertise

MediFind evaluates expertise by pulling from factors such as number of articles a doctor has published in medical journals, participation in clinical trials, speaking at industry conferences, prescribing and referral patterns, and strength of connections with other experts in their field.

Learn more about MediFind’s expert tiers

Find Dr. Harmatz's expertise for a condition
ConditionClose
View All 8 Experienced Conditions
Want to save this doctor for later?
Sign Up
Is this your doctor?
Find A Second Opinion
Not sure about your diagnosis?
Check Your Symptoms
 
 
 
 
Learn about our expert tiers
Learn More
Are you the provider on this profile?
Claim Profile