R. Douglas Wilson, Journal of Obstetrics and Gynaecology Canada, Available online 7 June 2016, ISSN 1701-2163, doi:10.1016/j.jogc.2016.04.091.
Most importantly this update is intended to help readers know when they need assistance from a reproductive geneticist for making decisions and providing choices.
TNR disorders are due to genomic instability/expansion. The effects of the TNR expansion are varied with loss of gene expression, a gain of gene function, or abnormal RNA processing. TNR disorders include conditions such as fragile X syndrome, myotonic dystrophy, Huntington disease, spinal bulbar muscular atrophy, and Friedreich ataxia (as well as other inherited ataxias)
Tuesday, June 21, 2016
Directions for new developments on statistical design and analysis of small population group trials
Ralf-Dieter Hilgers, Kit Roes, Nigel Stallard and for the IDeAl, Asterix and InSPiRe project groups. Orphanet Journal of Rare Diseases 201611:78 DOI: 10.1186/s13023-016-0464-5.
Open Access. Creative Commons Attribution 4.0 International License
This paper aims to raise awareness of the ongoing research and stimulate other groups to work on statistical methodology on design and analysis of clinical trials in small populations. Having well informed researchers, physicians and biostatisticians will result in the use of more efficient methods to conduct a clinical trial in small population groups and thus bring approved treatments faster to our patients.
Open Access. Creative Commons Attribution 4.0 International License
This paper aims to raise awareness of the ongoing research and stimulate other groups to work on statistical methodology on design and analysis of clinical trials in small populations. Having well informed researchers, physicians and biostatisticians will result in the use of more efficient methods to conduct a clinical trial in small population groups and thus bring approved treatments faster to our patients.
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