Publications & Presentations
Get key insights from published articles, posters, and other rare disease research from our team of clinicians and scientists.
14th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference
Phase 3 SELVA: Investigational QTORIN™ Sirolimus Topical Gel 3.9% Improved Disease Severity and Key Lesion Characteristics in Children With Microcystic Lymphatic Malformations
Lead Author: Maria Gnarra Buethe, MD, PhD, FAAD, Director of Pediatric and Adolescent Dermatology, University of California, Irvine
Lead Author: Maria Gnarra Buethe, MD, PhD, FAAD, Director of Pediatric and Adolescent Dermatology, University of California, Irvine
Phase 2 TOIVA: Overall Disease Severity and Lesion Bleeding Were Reduced in Pediatric Participants With Cutaneous Venous Malformations Treated With Investigational QTORIN™ Sirolimus Topical Gel 3.9%
Lead Author: Maria Gnarra Buethe, MD, PhD, FAAD, Director of Pediatric and Adolescent Dermatology, University of California, Irvine
Lead Author: Maria Gnarra Buethe, MD, PhD, FAAD, Director of Pediatric and Adolescent Dermatology, University of California, Irvine
26th Annual Fall Clinical Dermatology Conference
Patient- and Clinician-Reported Meaningfulness of Treatment-Related Change Following Investigational Sirolimus Topical Gel 3.9% in Microcystic Lymphatic Malformations: Qualitative Findings from the Phase 3 SELVA Trial Show Clinical Benefit and High Treatment Satisfaction
Lead Author: Maria Gnarra Buethe, MD, PhD, FAAD, Director of Pediatric and Adolescent Dermatology, University of California, Irvine
Lead Author: Maria Gnarra Buethe, MD, PhD, FAAD, Director of Pediatric and Adolescent Dermatology, University of California, Irvine
Systemic Pharmacokinetics of Investigational Sirolimus Topical Gel 3.9% Under Maximal Use Conditions in Microcystic Lymphatic Malformations: Results from the Phase 3 SELVA PK-MuST
Lead Author: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
Lead Author: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
Real world analysis of paid claims database reveals that disseminated superficial actinic porokeratosis (DSAP) is large orphan disease
Presenter: Abrar A. Qureshi, MD, MPH, Professor of Dermatology and Epidemiology, Brown University
Presenter: Abrar A. Qureshi, MD, MPH, Professor of Dermatology and Epidemiology, Brown University
International Society for the Study of Vascular Anomalies (ISSVA) World Congress 2026
QTORIN™ 3.9% Rapamycin Anhydrous Gel: Statistically Significant, Clinically Meaningful Improvement in Microcystic Lymphatic Malformations (Phase 3 SELVA Study) and Cutaneous Venous Malformations (Phase 2 TOIVA Study)
Presenter: James Treat, MD, Professor of Clinical Pediatrics and Dermatology at the Children’s Hospital of Philadelphia
Presenter: James Treat, MD, Professor of Clinical Pediatrics and Dermatology at the Children’s Hospital of Philadelphia
Society for Investigative Dermatology (SID) 2026 Annual Meeting
Significant reductions in lesion bleeding with QTORIN™ 3.9% rapamycin in cutaneous venous malformations
Presenter: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
Presenter: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
12th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference
SELVA: A Phase 3 Study with a Fit-For-Purpose Primary Endpoint Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
Presenter: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
Presenter: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
QTORINTM rapamycin 3.9% anhydrous gel: A Novel, Dermal-Targeted Topical mTOR Inhibitor in Phase 3 for Treatment of Microcystic Lymphatic Malformations
Presenter: Jeff Martini, Ph.D., Chief Scientific Officer, Palvella Therapeutics
Presenter: Jeff Martini, Ph.D., Chief Scientific Officer, Palvella Therapeutics
Rare Genodermatoses
Licholai G. Rare Dermatology: Orphan Disease Drug Development Opportunities and Challenges. Dermatology Summit (2019).
Doux J. Barriers and Opportunities: A View Across the Developmental Divide. Journal of Investigative Dermatology (2015) 135, 2143–2144.
Microcystic Lymphatic Malformations
Palvella Therapeutics, Inc. Microcystic and Macrocystic Lymphatic Malformations: Distinct Genetics and Clinical Strategies. Journal of Vascular Anomalies (2026).
Teng JMC, et. al., Sirolimus in the Treatment of Microcystic Lymphatic Malformations: A Systematic Review. Lymphatic Research and Biology (2022).
Carroll S, et. al., Annual prevalence estimation of lymphatic malformation with a cutaneous component: observational study of a national representative sample of physicians. Orphanet Journal of Rare Diseases (2022).
Canaud G, et al., A review of mechanisms of disease across PIK3CA-related disorders with vascular manifestations. Orphanet Journal of Rare Diseases (2021).
Adams DM, et al., Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies. Pediatrics (2016).
Hammill AM, et al., Sirolimus for the treatment of complicated vascular anomalies in children. Pediatric Blood and Cancer (2011).
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