Your Voice: Impact of DMD; a Qualitative Assessment of the Impact of DMD on Families

Toward patient-centered treatment goals for duchenne muscular dystrophy: insights from the “Your Voice” study burden of illness survey

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

Lifelong impact of ENPP1 Deficiency and the early onset form of ABCC6 Deficiency from patient or caregiver perspective

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

Engage’s President and CEO, Patti Engel, is a patent-holder on the Xyrem® risk management program

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

Patient Reported Outcome Data from Acromegaly Patients Treated with Injectable Somatostatin Analogues in Routine Clinical Practice: Preliminary Results

Biochemically Controlled Acromegaly Patients on a Stable Dose of Injectable Somatostatin Analogues (SSAs) in Routine Clinical Practice Still Remain Symptomatic: Preliminary Results from Endocrinology Health Care Professionals

Relationship between Responses from Acromegaly Patients Treated with a Stable Dose of Injectable Somatostatin Analogues in Routine Clinical Practice and their Endocrinology Health Care Professional regarding treatment Outcomes: Preliminary Findings

Enhancing Awareness and Understanding of Gene Therapy among Rare Disease Communities: A Research-Driven Roadmap

Physician and Patient Perceptions Regarding Physician Training in Rare Diseases: The Need for Stronger Educational Initiatives for Physicians

The Role of Patient Advocacy in Drug and Device Development for Rare Diseases