Paul Harmatz

Professor in Residence

University of California, San Francisco

United States

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About

Paul Harmatz is a Professor in Residence at the University of California San Francisco. His research primarily focuses on the long-term efficacy and safety of treatments for rare genetic diseases, including Niemann-Pick disease and mucopolysaccharidoses. He has contributed to significant clinical studies regarding enzyme replacement therapy and gene therapy, with a particular emphasis on improved patient outcomes. His work also encompasses the safety of anesthesia in patients with these conditions.

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2023

Collaborators (3)

Igor Nestrasil

University of Minnesota

UNITED STATES

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Adriana Montaño

Saint Louis University

UNITED STATES

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Tariq Aslam

The University of Manchester

UNITED KINGDOM

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Paul Harmatz

Research Interests

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About

Articles (30)

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 enzyme replacement therapy: Findings from the mucopolysaccharidosis VI clinical surveillance program after 15 years follow-up

Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Safety of anesthesia in mucopolysaccharidoses - A comparative retrospective cohort study on more than 600 cases

Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase‐IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II

Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study

Oral Infigratinib Therapy in Children with Achondroplasia

A phase 1/2 study of LY3884961 (PR001) an AAV9-based gene therapy for Gaucher disease type 2 – A clinical update from the PROVIDE trial

Anesthesia in mucopolysaccharidoses – Retrospective analysis and insights of a large cohort

Application of a natural history study on an investigational drug clinical development

Biomarkers of Glycosaminoglycans (GAG) accumulation in patients with mucopolysaccharidosis type VI-LeukoGAG, Corneal Opacification (COM) and Carotid Intima Media Thickening (CIMT)

In Utero Enzyme-replacement Therapy for Infantile-onset Pompe Disease

CAMPSIITE™ Phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)

α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial

A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: Updated study design

A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Baseline clinical data

A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Patients' and caregivers' assessments

A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidosis (PRONTO): Evaluation of different assessments

CAMPSIITE™ phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)

Design of a Multi-Center Randomized Active Controlled Phase 3 Clinical Trial (HURCULES) Evaluating the Safety and Efficacy of OTL-203 in Patients with MPS-IH Versus Standard of Care with Allogeneic Hematopoietic Stem Cell Transplantation

A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

Growth patterns in patients with mucopolysaccharidosis VII

Corrigendum to: Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study

Corrigendum to “Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II”

Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10‐year prospective study

In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease

Current and new therapies for mucopolysaccharidoses

Interim analysis of key clinical outcomes from a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II

Interim results of a phase 1/2 study of JR-171 (lepunafusp alfa), a novel brain-penetrant enzyme replacement therapy for MPS I

RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human study

Collaborators (3)

Igor Nestrasil

Adriana Montaño

Tariq Aslam

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