About

Bill Byrne is an internationally exhibited multimedia digital artist, professor, academic leader, textbook author, animator, game artist, and motion graphics designer. He has written three textbooks for Focal Press: '3D Motion Graphics For 2D Artists' published in December 2012, 'Creative Motion Graphic Titling' (co-authored with Yael Braha) published in October 2010, and 'The Visual Effects Arsenal' published in April 2009. Byrne has been a college professor for seventeen years and served as the Academic Director of Media at the Art Institute of Austin from 2010 to 2016, overseeing all media programs and working directly on curriculum development for Animation, Game Art, Visual Effects, and Film. In 2019, he joined the faculty in the Arts and Entertainment Technologies department at the University of Texas at Austin. As a professional motion graphics designer, he has collaborated with clients such as Electronic Arts, NBC, Tiffany & Co., ABC Sports, ESPN, Panasonic, Snickers, and RCA Records. Byrne holds a Master of Fine Arts in Photography and Related Media from New York’s School of Visual Arts and is an Adobe Certified Expert in Photoshop. Born in Staten Island, New York, he now resides in Austin, Texas with his wife Suzanne and their three daughters, Elinor, Audrey, and Vivian.

Research topics

• Medicine
• Internal medicine
• Biology
• Virology
• Radiology
• Genetics
• Pathology
• Cell biology
• Cardiology
• Chemistry
• Biochemistry

Selected publications

• Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease

  Therapeutic Advances in Rare Disease · 2026-02-26

  articleOpen access

  Late-onset Pompe disease (LOPD) is a rare inherited disorder caused by deficiency of the lysosomal enzyme acid α-glucosidase (GAA), leading to an accumulation of lysosomal glycogen in tissues, profoundly affecting muscles. Patients with LOPD typically have some residual GAA activity but experience progressive skeletal muscle dysfunction resulting in muscle weakness and respiratory failure. Enzyme replacement therapy (ERT) with alglucosidase alfa, a recombinant human GAA (rhGAA), was the first disease-specific therapy for Pompe disease. Despite efficacy in the first years of use, many patients receiving alglucosidase alfa experience a decline in function over time. This may reflect the inherent challenges associated with rhGAA ERT, such as enzyme inactivation at the near-neutral pH of blood, inefficient target cell uptake, and a necessity for complete lysosomal processing once inside target cells. Cipaglucosidase alfa, a second-generation rhGAA, aims to address these challenges through natural enrichment with bis-mannose-6-phosphate-containing N -glycans to enhance cellular uptake while retaining capacity for complete postdelivery processing. Co-administration of cipaglucosidase alfa with the small molecule stabilizer miglustat (N-butyldeoxynojirimycin) enhances cipaglucosidase alfa stability in the bloodstream after infusion. We discuss published and new preclinical and clinical data on the efficacy and safety of miglustat in combination with cipaglucosidase alfa for treating LOPD. Studies in Pompe mouse models and patients with Pompe disease showed that stabilization by miglustat improved cipaglucosidase alfa exposure and availability for uptake into target tissues and was associated with improved functional outcomes and biomarker levels compared with cipaglucosidase alfa alone. In patients with Pompe disease, the once every 2 weeks dosing regimen of miglustat was well tolerated, with a low frequency of miglustat-related gastrointestinal events compared with daily miglustat regimens at higher doses used in the treatment of other diseases. Trial registration : New data are reported for NCT02675465 (ATB200-02), NCT03729362 (PROPEL), and NCT04138277 (PROPEL open-label extension, ATB200-07); all registered at ClinicalTrials.gov ( https://clinicaltrials.gov ).

  PublisherDOI

• Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials

  Therapeutic Advances in Rare Disease · 2026-01-01

  articleOpen accessSenior author

  Background: Late-onset Pompe disease (LOPD) is caused by a deficiency of the acid α-glucosidase enzyme. In LOPD treatment, enzyme replacement therapy is delivered via intravenous infusion, typically in clinical settings. Cipaglucosidase alfa is delivered with the oral enzyme stabilizer miglustat (cipa + mig). Objectives: Evaluate the safety of cipa + mig home infusions. Design: Post hoc analysis of pooled safety data from three clinical trials in adults with LOPD (NCT02675465, NCT03729362, NCT04138277). Methods: The frequency and severity of infusion-associated reactions (IARs) during cipa + mig home and clinic administration were analyzed. Results: In total, 65/151 patients (43.0%) received ⩾1 cipa + mig home treatment. Of 9185 treatments, 2024 (22.0%) were administered at home. IAR frequency was similar for home (1.3%, 26/2024) and clinic (1.8%, 129/7161). The most frequent IAR following home infusion was pyrexia (6.2% of patients). Two patients with ⩾1 home-based treatment experienced serious IARs. Conclusion: Analyses support the safety of home cipa + mig treatment in eligible adults with LOPD.

  PublisherDOI

• 90-month physician and patient-reported outcomes with cipaglucosidase alfa plus miglustat (cipa + mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study

  Molecular Genetics and Metabolism · 2026-02-01

  article
  PublisherDOI

• 90-month muscle function and biomarker outcomes with cipaglucosidase alfa plus miglustat (cipa + mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study

  Molecular Genetics and Metabolism · 2026-02-01

  article
  PublisherDOI

• Thorough QT Study on the Effect of Therapeutic and Supratherapeutic Dosing of Givinostat in Healthy Volunteers

  Clinical Pharmacology in Drug Development · 2026-03-01

  articleOpen access

  Abstract Givinostat is a class I/II histone deacetylase inhibitor indicated for Duchenne muscular dystrophy (DMD). The study evaluated the effect of therapeutic and supratherapeutic givinostat doses on the QT/QTc interval. Healthy volunteers received each treatment—givinostat hydrochloride monohydrate oral suspension as a therapeutic (100 mg) or supratherapeutic (300 mg) dose, placebo oral suspension, or moxifloxacin oral tablet (positive control, 400 mg)—according to a block randomization scheme. Cardiodynamic assessments were paired with pharmacokinetic samples. A small, clinically non‐relevant effect on mean placebo‐corrected, change‐from‐baseline QTcF (∆∆QTcF) of 5.5 ms was seen after givinostat 100‐mg dose. Clinically relevant QTc prolongation was observed with the supratherapeutic dose, with a mild ∆∆QTcF increase of 13.6 ms. A delay of ≈3 h between T max and the largest effect on the QTc interval was seen for both doses. In the concentration‐QTc analysis, an E max model captured the data better than the prespecified linear model and showed that an effect on ∆∆QTcF exceeding 10 ms could be excluded within the full range of observed givinostat concentrations in this study and up to ≈745 ng/mL. Givinostat at the maximum labeled dose (up to 53.2 mg twice daily for DMD) is not expected to pose a QT prolongation risk.

  PublisherDOI

• 90-month pulmonary function outcomes with cipaglucosidase alfa plus miglustat (cipa + mig) in adults with Pompe disease in ATB200-02, an open-label phase I/II study

  Molecular Genetics and Metabolism · 2026-02-01

  articleSenior author
  PublisherDOI

• P169: 90-month efficacy outcomes with cipaglucosidase alfa plus miglustat in adults with Pompe disease in ATB200-02: An open-label phase I/II study

  Genetics in Medicine Open · 2026-01-01

  articleOpen access

  homocysteine levels or vitamin B12 levels in All of Us (data not available for UK Biobank).Prior associations between MTHFR thermolabile variant heteroor homozygosity and other phenotypes such as thrombosis, stroke, cardiovascular disease, cancer, or congenital malformations of the nervous system were not replicated in this study.Conclusion: Our data showed no significant corroboration of previously linked common conditions including thrombosis, stroke, cardiovascular disease, cancer, or congenital malformations of the nervous system to MTHFR c.665C>T genotypes in these large datasets.These findings strongly support recommendations to not test individuals for MTHFR c.665C>T.

  PublisherOA PDFDOI

• AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial

  New England Journal of Medicine · 2026-02-06 · 1 citations

  article

  BACKGROUND: , results from deficiency of lysosomal β-galactosidase, which degrades GM1 ganglioside. This fatal neurodegenerative disease currently has no effective therapy. METHODS: In a phase 1-2, open-label, dose-escalation study, we assessed immunosuppression and a single intravenous infusion of adeno-associated virus serotype 9 (AAV9) encoding β-galactosidase in children with type II GM1 gangliosidosis with late-infantile or juvenile onset. The primary end point was safety. Secondary end points included changes from baseline in the cerebrospinal fluid (CSF) GM1 ganglioside concentration and β-galactosidase activity, clinical assessments (including the Clinical Global Impression-Improvement [CGI-I] score, assessed on a scale from 1 [very much improved] to 7 [very much worse]), and neuroimaging patterns. RESULTS: Nine participants were enrolled. Over a 3-year period, 124 adverse events occurred, 30 of which (8 gastrointestinal events, 21 laboratory abnormalities associated with inflammation, and 1 tachycardia event) were deemed by the investigator as being possibly, probably, or definitely related to the gene therapy. Five serious adverse events occurred, including vomiting that led to hospitalization in one participant, which was attributed to the gene therapy. Serum aspartate and alanine aminotransferase levels increased in all participants and returned to baseline levels by 18 months. In all participants, the CSF β-galactosidase level increased and CSF GM1 ganglioside level decreased. Expressive communication and gross motor scores appeared stable, but fine motor and receptive communication scores decreased. The median CGI-I score was 3 (indicating minimal improvement) at 2 years and 4 (indicating no change) at 3 years; in historical controls, scores have been shown to increase (indicating worsening) over time. Neuroimaging showed patterns consistent with reduced rates of cerebral atrophy and favorable changes in myelination as compared with baseline. CONCLUSIONS: In this study involving nine participants with type II GM1 gangliosidosis, a single infusion of AAV9 encoding β-galactosidase was associated with adverse events, including severe vomiting in one participant and elevated liver-enzyme levels in all participants. Secondary end-point results suggested improvements in biochemical markers and neuroimaging patterns and stable or reduced rates of developmental deterioration in some measures. (Funded by the National Human Genome Research Institute and others; ClinicalTrials.gov number, NCT03952637.).

  PublisherDOI

• Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRI

  Translational research · 2026-02-19

  articleOpen access

  Pompe disease is a glycogen storage disease caused by the impaired breakdown of glycogen in lysosomes, leading to abnormal glycogen accumulation in tissue. Here we use glycogen nuclear Overhauser effect (glycoNOE) MRI to detect glycogen levels in skeletal muscle in a mouse model of Pompe disease. Moreover, we evaluated if glycoNOE MRI could detect changes in glycogen load after enzyme replacement therapy. The results show that glycoNOE MRI can distinguish between Pompe mice and wildtype controls. Furthermore, the technique detected treatment-dependent changes in muscle glycoNOE signals, which were validated with ex vivo biochemical assays. To demonstrate potential human translation, glycoNOE MRI was applied to two Pompe patients and revealed elevated glycogen levels in patients compared to healthy controls.

  PublisherDOI

• Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease

  Molecular Therapy · 2025-06-14 · 2 citations

  articleOpen accessSenior author
  PublisherOA PDFDOI

Recent grants

• Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease

  NIH · $1.6M · 2015–2021

• NIH Grant P50HL059412

  NIH · $13.9M · 2002

• NIH Grant M01RR000082

  NIH · $30.1M · 2011

• NIH Grant P01HL051811

  NIH · $38.5M · 2015

• NIH Grant R01DK058236

  NIH · $424k · 2003

Frequent coauthors

• Richard S. Finkel

  St. Jude Children's Research Hospital

  510 shared

• Craig M. McDonald

  UC Davis Health System

  448 shared

• Alberto Dubrovsky

  Favaloro University

  443 shared

• Nathalie Goemans

  KU Leuven

  443 shared

• Haluk Topaloğlu

  441 shared

• Krista Vandenborne

  University of Florida

  275 shared

• H. Lee Sweeney

  University of Florida

  271 shared

• Ronald G. Victor

  Maputo Central Hospital

  260 shared