Network meta-analysis is a popular method to synthesize the information obtained in a systematic review of studies (e.g., randomized clinical trials) involving subsets of multiple treatments of interest. The dominant method of analysis employs within-study information on treatment contrasts and integrates this over a network of studies. One…

The peroxisome biogenesis disorders (PBD) are a heterogeneous group of autosomal recessive disorders in which peroxisome assembly is impaired, leading to multiple peroxisome enzyme deficiencies, complex developmental sequelae and progressive disabilities. Mammalian peroxisome assembly involves the protein products of 16 "PEX" genes;…

Researchers in the medical domain consider the double-blind placebo controlled clinical trial the gold standard. The data for these clinical trials are collected for a specifically defined hypothesis and there is very little in the realm of secondary data analyses conducted. The underlying purpose of this work is to demonstrate the value and…

Of the nearly 7,000 rare diseases identified by the National Institutes of Health (NIH), only a few hundred currently have treatments. The development of therapies for rare diseases is often hampered by the special challenges of conducting the needed studies for rare disease drugs and medical devices, such as small numbers of patients and the fact…