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Empowering the GM1 and GM2 community with a co-created education resource about a natural history study
by
Lewi, Daniel
,
Pangonis, Diana
,
Stokes, Louis
,
Cognitant, Alex M.
,
Linthorst, Gisela
Published in
Molecular genetics and metabolism
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Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: N...
by
Fields, Taylor
,
Crowe, Joslyn
,
Evans, Will
,
Greenfield, Julie
,
Hopkin, Justin
,
Thornton, Jennifer K.
,
Lewi, Daniel
,
Mathieson, Toni
,
Pangonis, Diana
Published in
Molecular genetics and metabolism
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GM1‐gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat
by
Bingaman, Amanda
,
Waggoner, Christine
,
Andrews, Sara M.
,
Pangonis, Diana
,
Trad, Marie
,
Giugliani, Roberto
,
Giorgino, Ruben
,
Jarnes, Jeanine
,
Vakili, Rojan
,
Ballard, Victoria
,
Peay, Holly L.
Published in
American journal of medical genetics. Part A
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Molecular Genetics And Metabolism
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American Journal Of Medical Genetics. Part A
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