A Aralex Aachen research explorer

Company ROR OpenAlex

Pharming Group (Netherlands) Sign in to save

45 stored papers · Search its papers → · Its authors →

Papers 45

Recombinant Human C1 Esterase Inhibitor Treatment in Addition to Standard of Care for the Prevention of Cytokine Storm in Hospitalized Patients with COVID-19: A Randomized, Open-Label Trial
2022 SSRN Electronic Journal preprint Medicine COVID-19 Clinical Research Studies Open access

0

citations
EE39 Costs of Treating Hereditary Angioedema (HAE) Attacks with C1 Inhibitors in Four European Countries
2022 Value in Health conference-abstract Medicine Coagulation, Bradykinin, Polyphosphates, and Angioedema Open access

0

citations
EE681 Costs of Treating Acute Hereditary Angioedema (HAE) Attacks With C1 Inhibitors in Five Central and Eastern European Countries
2022 Value in Health conference-abstract Medicine Coagulation, Bradykinin, Polyphosphates, and Angioedema Open access

0

citations
Analyse intermédiaire de la tolérance et des paramètres hématologiques de patients atteints du syndrome APDS traités par lénionisib, inhibiteur de PI3Kδ, dans une étude d’extension ouverte à long terme
2023 La Revue de Médecine Interne article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders

0

citations
PCR30 Benefits and Challenges of Different Qualitative Approaches in Rare Disease: A Case Study in Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Using a Narrative Exercise and Qualitative Interviews
2023 Value in Health conference-abstract Social Sciences Focus Groups and Qualitative Methods Open access

0

citations
HTA25 What Is a Highly Specialised Technology (HST)? The Revised NICE HST Criteria in Practice
2023 Value in Health conference-abstract Economics, Econometrics and Finance Health Systems, Economic Evaluations, Quality of Life Open access

0

citations
177 ClinGen Framework for PIK3CD Variant Classification: Use of Adapted ACMG/AMP Guidelines
2024 Clinical Immunology article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases

0

citations
32 Clinical and Genetic Findings of >5,300 Individuals Tested via the navigateAPDS Sponsored Genetic Testing Program
2024 Clinical Immunology article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations
57 Development and Implementation of the TRIO Health Activated Phosphoinositide 3-kinase Delta Syndrome Characterization and Clinical Outcomes Immunologic Registry (APDS-CHOIR)
2024 Clinical Immunology article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders

0

citations
Deuxième analyse intermédiaire d’une étude en ouvert du Leniolisib dans le syndrome PI3Kδ activée : efficacité et tolérance long terme
2024 La Revue de Médecine Interne article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders

0

citations
Leniolisib treatment for people with activated PI3K delta syndrome (APDS): a plain language summary of the phase 3 study
2024 Future Rare Diseases article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations
HTA366 Challenges and Criteria for Accepting Lower-Level of Evidence in Early Benefit Assessments (EBA) in Germany
2024 Value in Health conference-abstract Economics, Econometrics and Finance Health Systems, Economic Evaluations, Quality of Life Open access

0

citations
The BeWayAPDS project: Designing the best way to optimize management of activated PI3Kδ syndrome (APDS) in Spain from the perspective of key stakeholders
2025 Rare article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations
Primary and Safety Outcomes of a Phase 3 Open-Label, Single-Arm, 12-Week Study of Treatment with PI3Kδ Inhibitor Leniolisib in Pediatric Patients Aged 4–11 Years with Activated PI3Kδ Syndrome (APDS)
2025 Journal of Human Immunity article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations
Patient Insights Expand Understanding of APDS
2025 Journal of Human Immunity article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations
Demographic and Clinical Characteristics of People with Activated Phosphoinositide 3-Kinase Delta Syndrome in the APDS Characterization and Clinical Outcomes Immunologic Registry
2025 Journal of Human Immunity article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations
CO12 APDS Treatment in Germany: Decision Criteria, Costs, and Contribution of Leniolisib to Outcomes
2025 Value in Health conference-abstract Immunology and Microbiology Immunodeficiency and Autoimmune Disorders

0

citations
EE737 Treatment Patterns of Rare Immunodeficiencies With Special Regard to Antibiotics: A German Claims Data Analysis
2025 Value in Health conference-abstract Immunology and Microbiology Immunodeficiency and Autoimmune Disorders

0

citations
Discovery, α-Glucosidase Inhibitory Activity, and Structural Optimization of the Alkaloids from Vanderbylia robiniophila SY636
2026 Journal of Natural Products article Medicine Fungal Biology and Applications

0

citations
Caregivers of Children with APDS Balance Complex Medical Care, Family, and Work Responsibilities
2026 Journal of Human Immunity article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access

0

citations

Most cited authors