Health Insurance Literacy in Acid Sphingomyelinase Deficiency (ASMD)

Fear Related to Access Issues in Acid Sphingomyelinase Deficiency (ASMD)

Understanding the Functional Burden of CLN3 Through the Eyes of Patients and Families

Patient versus caregiver burden in ASMD

The impact of fibrodysplasia ossificans progressiva (FOP) on patients and their family members: results from an international burden of illness survey

Patient Voice in Access Studies

Health insurance literacy and health services access barriers in Niemann–Pick disease: the patient and caregiver voice

GACI and ENPP1 Publication

Enabling Precision Medicine Trials: A Success Story in a Movement Disorder Setting

Health Care Access satisfaction and quality of life among children and adults with rare diseases

Engage Health, Inc., Methodologies for Conceptual Strength Regarding the Burden of Access

Abstract Regarding Engage Health’s Burden of Access in Niemann-Pick Disease Study Accepted for Presentation at the 17th Annual WORLDSymposium

Rare Disease Qualitative Research: Methodologies for Conceptual Strength and Representativeness

The challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers

Observed discordance between outcomes reported by acromegaly patients and their treating endocrinology medical provider