Rare Disease

Emmes experts discuss the challenges and solutions related to a lack of insight into a patient's diagnosis and care journey

Rare diseases present unique challenges in drug development due to their low prevalence and the diverse nature of each individual condition

Pediatric trials for rare diseases are crucial. We discuss the complexities of agreeing the definitions and approaches used to measure trial success that are meaningful for all involved

Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregivers

Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregivers

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Moving beyond standard endpoints in rare disease clinical trials has never been more crucial. Explore five areas of meaningful, patient centered assessment and innovative strategies that drive impactful results for improving the lives of patients and resh

Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia

Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia

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Assessing Psychosocial Readiness for Gene Therapy in Sickle Cell Disease: A CureSCi Consensus Statement

Assessing Psychosocial Readiness for Gene Therapy in Sickle Cell Disease: A CureSCi Consensus Statement

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Rare Disease

Immune Thrombocytopenic Purpura (ITP) - Rare Disease and Pediatric Center

Implementation Science and Examples of Emmes’ Support in Opioid Use Disorder

The Rare Disease Patient Experience

Emmes experts discuss the challenges and solutions related to a lack of insight into a patient's diagnosis and care journey

Rare diseases present unique challenges in drug development due to their low prevalence and the diverse nature of each individual condition

Pediatric trials for rare diseases are crucial. We discuss the complexities of agreeing the definitions and approaches used to measure trial success that are meaningful for all involved

Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregivers

Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregivers

Moving beyond standard endpoints in rare disease clinical trials has never been more crucial. Explore five areas of meaningful, patient centered assessment and innovative strategies that drive impactful results for improving the lives of patients and resh

Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia

Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia

Assessing Psychosocial Readiness for Gene Therapy in Sickle Cell Disease: A CureSCi Consensus Statement

Assessing Psychosocial Readiness for Gene Therapy in Sickle Cell Disease: A CureSCi Consensus Statement