Melissa Hogan

Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction.

Yow-Ming C Wang, Anne R. Pariser, J. Bluestone, 2016, Molecular Genetics and Metabolism.

(R)evolution: toward a new paradigm of policy and patient advocacy for expanded access to experimental treatments

Melissa Hogan, 2016, BMC Medicine.

Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints.

J. H. van der Lee, J. Eisengart, R. Giugliani, 2020, Molecular genetics and metabolism.

Toileting Abilities Survey as a surrogate outcome measure for cognitive function: Findings from neuronopathic mucopolysaccharidosis II patients treated with idursulfase and intrathecal idursulfase

C. Hendriksz, L. Edwards, Kim Stephens, 2020, Molecular Genetics and Metabolism Reports.

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure.

J. H. van der Lee, Darren T. Janzen, R. Giugliani, 2017, Molecular genetics and metabolism.

Backpack health reduces data‐sharing barriers between the medical community and individuals with rare diseases

H. LaDuca, M. Towne, Lauren May, 2020, American journal of medical genetics. Part C, Seminars in medical genetics.

Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions.

J. Mink, F. Eichler, S. Waisbren, 2016, Molecular genetics and metabolism.