Clinical Compendium - PANTHERx Rare

Publications

Advancing rare disease care through research & education

Discover the expertise and innovation of our clinical pharmacy team through published research, posters, abstracts, articles, and continuing education (CE) presentations.

April 20, 2026

The Complexity of Care: A Comprehensive Look at Prader-Willi Syndrome from Symptoms to Emerging Therapies.

April 17, 2026

Evaluating the Impact of Dedicated Clinicians on Prior Authorization Outcomes and Access to Therapy in Rare Diseases

April 17, 2026

Impact of Quick Start and Bridge Programs on Rare Disease Therapy Initiation and Preventing Gaps in Therapy Objective

April 17, 2026

The Impact of Team Member Engagement on Quality Metrics, Process Improvements and Patient Safety

March 27, 2026

The Rare Journey Needs a Special Pharmacy Partner

March 10, 2026

Genetic Characterization and Clinical Manifestations in Adults with Hypophosphatasia in the United States

March 2, 2026

Specialty Pharmacy Continuum: As Rare Disease Drug Approvals Accelerate, Survey Highlights Gaps in Care Coordination

February 17, 2026

From Hunger to Harmony: Neuroendocrine Pathways and Therapeutic Strategies in Metabolic Disorders Associated with Hyperphagia

January 9, 2026

Targeted Reach, Tighter Relationships: Why Rare Disease Launches Are Different

December 11, 2025

Muscle Matters: Pathophysiology, Diagnosis, and Treatment of Duchenne Muscular Dystrophy

November 7, 2025

Medication Adherence in Wilson Disease Patients Treated with Trientine Tetrahydrochloride Supplied by a Single-Source Specialty Pharmacy

November 6, 2025

Real-World Adherence and Persistence with Leniolisib in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

November 6, 2025

Effectiveness of Leniolisib in Reducing Infections Among Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

October 17, 2025

Hyaluronidase 101: From Science to Clinical Applications

September 12, 2025

New article from PANTHERx CEO, Bansi Nagji, explores how hyper-personalization is reshaping the way we approach rare disease care

July 31, 2025

Shaping the Future of Generalized Myasthenia Gravis Management: A Focus on Novel Treatment Approaches

March 31, 2025

The Impact of Patient Familiarity with their Pharmacy Care Team on Medication Adherence and Communication Effectiveness in Disease Management

March 31, 2025

Evaluating the Impact of Enhanced Cooler Return Services for Rare Cold Chain Medications Based on Environmental, Financial, and Patient Perspectives

March 31, 2025

The Impact of Telephonic and Printed Patient Counseling Measures on Adverse Events and Medication Discontinuation

March 20, 2025

Assessing Medication Adherence in Wilson Disease Patients Treated with Trientine Tetrahydrochloride Supplied by a Single-Source Specialty Pharmacy

February 7, 2025

Today’s Rare Disease Launches Require the Right Specialty Pharmacy Partner

December 19, 2024

The Impact and Role of Pharmacists in the Management of Specialty Therapies

December 19, 2024

Strategies to Reduce Challenges in the Management of Chronic Rhinosinusitis with Nasal Polyps

December 19, 2024

Optimizing Treatment Strategies: A Comprehensive Approach to Primary Biliary Cholangitis and Pruritus

October 29, 2024

Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan

October 9, 2024

Beyond the Surface: Specialized Insights into Epidermolysis Bullosa

April 15, 2024

The Impact of Enhanced Two-Way Text Services and Digital Services on Adherence in the Rare Disease Population

April 15, 2024

Factors Associated with Prescription Abandonment and Non-Persistence within Six Months of Initiating Oral First-In-Class Rare Disease Medications

May 17, 2023

Evolving Treatment Landscape of Rett Syndrome: Emerging Therapies to Address Critical Clinical Gaps

March 21, 2023

The Impact of Site of Service on Patient Outcomes with Healthcare Professional Administered Specialty Medications

March 21, 2023

The Impact of Scheduled Telephonic Clinician Intervention on Medication Adherence and Persistence in Rare Disease Patients

November 15, 2022

Early Experience and Patient Characteristics from Opera: A Real-World Study of Pegcetacoplan Treatment

October 7, 2022

A Rare Specialty Pharmacy’s Quick Response to Customized Patient Education Using QR Codes

May 16, 2022

Abstract: A Retrospective Analysis to Assess the Effect of Targeted Clinical Interventions on the Discontinuation Rate of Amikacin Liposome Inhalation Suspension Therapy among Patients Treated for Refractory Mycobacterium Avium Complex Lung Disease

April 13, 2022

Exploring Newer Treatment Options in ANCA-Associated Vasculitis to address Management Gaps

March 29, 2022

Retrospective Analysis to Assess the Impact of the Use of a Medication-Adherence Smart Bottle on Clinical Outcomes in a Rare Disease Population

April 12, 2021

Retrospective Analysis to Assess the Discontinuation of Amikacin Liposome Inhalation Suspension Therapy Among Patients Treated for Refractory Mycobacterium Avium Complex Lung Disease

April 12, 2021

A Retrospective Analysis to Assess the Effect of Targeted Clinical Interventions on the Discontinuation Rate of Amikacin Liposome Inhalation Suspension Therapy among Patients Treated for Refractory Mycobacterium Avium Complex Lung Disease

April 12, 2021

Assessing the Knowledge and Perceptions of Healthcare Providers on Motivational Interviewing and the Impact on Patient Adherence in Rare Disease Management

April 21, 2020

Assessment of Primary Medication Nonadherence for Newly Referred Valbenazine Patients and Corresponding Barriers to Receiving Therapy

March 25, 2019

Retrospective Analysis of Patient-Specific Differences Between Patients on Valbenazine 40mg Once Daily and 80mg Once Daily

March 25, 2019

Targeted Specialty Medication Referral Form Updates, How Do They Impact Patients and Providers

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