Concepedia

A master protocol to investigate a novel therapy acetyl-l-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia

Taylor Fields, Marc C. Patterson, Tatiana Brémovà-Ertl, G Belcher, Ian Billington, Grant C. Churchill, W. Davis, William Evans, Stephen Flint, U. Granzer,

Trials · 2021 · 34 citations · 23 references

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References

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The Columbia–Suicide Severity Rating Scale: Initial Validity and Internal Consistency Findings From Three Multisite Studies With Adolescents and Adults

Kelly Posner, Gregory K. Brown, Bárbara Stanley et al. · American Journal of Psychiatry · 2011 · 4.8K citations

Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both

Janet Woodcock, Lisa M. LaVange · New England Journal of Medicine · 2017 · 926 citations · Full text

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Linear clinical progression, independent of age of onset, in Niemann–Pick disease, type C

Nicole M. Yanjanin, Jorge I. Vélez, Andrea Gropman et al. · American Journal of Medical Genetics Part B Neuropsychiatric Genetics · 2009 · 197 citations

Visual gait analysis: the development of a clinical assessment and scale

S. Lord, Peter W. Halligan, Derick T Wade · Clinical Rehabilitation · 1998 · 190 citations

Disease and patient characteristics in NP-C patients: findings from an international disease registry

Marc C. Patterson, Eugen Mengel, Frits A. Wijburg et al. · Orphanet Journal of Rare Diseases · 2013 · 190 citations · Full text